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En esta sección de la página Internet de la FMH, los fabricantes de productos de tratamiento pueden publicar comunicados de prensa de interés para la comunidad mundial de trastornos de la coagulación.

La información aquí publicada de ninguna manera representa el respaldo de la FMH a un producto específico. Las declaraciones y opiniones expresadas aquí no necesariamente representan las opiniones, políticas o recomendaciones de la FMH.

Lista de los comunicados de prensa más recientes están disponibles únicamente en inglés.

Sar Therapeutics Announces Oversubscribed $125 Million Series D Financing
September 30, 2025 — Star Therapeutics Announces Oversubscribed $125 Million Series D Financing. Proceeds will go to support the continued clinical advancement of the lead program, VGA039, a first-in-class antibody targeting protein S for the treatment of bleeding disorders, starting with von Willebrand disease (VWD). VGA039 is the first subcutaneous therapy, dosed once monthly, that can address all types of VWD and all types of bleeds. A pivotal Phase 3 trial of VGA039 in VWD patients has been initiated. LINK


U.S. FDA approves expanded indication for von Willebrand factor (Recombinant) for adults and children with von Willebrand disease
September 5, 2025 — Takeda announced that the U.S. Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (sBLA) for von Willebrand factor (Recombinant), expanding the indication to include routine prophylaxis to reduce the frequency of bleeding episodes in adults with von Willebrand disease (VWD), including those with Type 1 and 2 disease, and on-demand and perioperative management of bleeding in pediatric patients with VWD. Von Willebrand factor (Recombinant) was previously approved for on-demand and perioperative use in adults with VWD and routine prophylactic use in adults with severe Type 3 VWD receiving on-demand therapy. LINK


FDA approves concizumab-mtci to prevent or reduce the frequency of bleeding episodes for hemophilia
July 31, 2025 — The US Food and Drug Administration (FDA) has approved concizumab-mtci as a once-daily prophylactic subcutaneous injection treatment to prevent or reduce the frequency of bleeding episodes for adults and children 12 years of age and older with hemophilia A or B without inhibitors. LINK


Pfizer announces positive topline Phase 3 Results for marstacimab in hemophilia A or B with inhibitors
June 26, 2025 — Pfizer Inc. has announced positive topline results from the Phase 3 BASIS study evaluating marstacimab for adults and adolescents living with hemophilia A or B with inhibitors. The study met the primary endpoint and key secondary bleeding endpoints demonstrating the superiority of once-weekly subcutaneous marstacimab injections in improving key bleeding outcomes compared to on-demand treatment in a patient population where less burdensome treatment approaches are needed. LINK


NXT007 may have potential to provide hemostatic normalization in people with hemophilia A
June 23, 2025 — Roche announced today positive phase I/II data on NXT007—a next-generation bispecific antibody—in people with hemophilia A, supporting its progression into phase III clinical development. Early data from the NXTAGE study suggest that NXT007 may have the potential to provide hemostatic normalization in people with hemophilia A. This positive phase I/II data was presented at the 2025 International Society on Thrombosis and Haemostasis (ISTH) Congress. LINK


Belief BioMed and Takeda China jointly announce official approval of Dalnacogene Ponparvovec Injection, China’s first hemophilia B gene therapy
April 10, 2025 — The World Federation of Hemophilia (WFH) is currently following a recent gene therapy development in China. Belief BioMed and Takeda China have jointly announced that Dalnacogene Ponparvovec Injection has been officially approved by the National Medical Products Administration (NMPA) for the treatment of adult patients with moderate to severe hemophilia B (congenital coagulation factor IX deficiency) in China. The WFH will keep our community informed as new developments are shared. LINK

To read the ASH Publications article, “Efficacy and Safety of an Engineered, Liver-Tropic Adeno-Associated Virus Vector Expressing Factor IX Padua Administered with Prophylactic Glucocorticoids in Patients with Hemophilia B: A Multi-Center, Single-Arm, Phase 3 Trial”, please click here.


Fitusiran approved as the first therapy in the U.S.A to treat hemophilia A or B with or without inhibitors
March 28, 2025 — The US Food and Drug Administration (FDA) has approved Fitusiran, the first antithrombin-lowering (AT) therapy for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients (aged 12 or older) with hemophilia A or B with or without factor VIII or IX inhibitors. LINK


Pfizer stops commercialization of fidanacogene elaparvovec-dzkt gene therapy
February 20, 2025 — Pfizer will stop global development and commercialization of fidanacogene elaparvovec-dzkt, a gene therapy treatment, because of low demand from patients and physicians. The treatment is a one-time therapy that was approved in in several countries— including the U.S.A, the E.U, Canada and Japan—for the treatment of adults with moderate to severe hemophilia B. Pfizer will redirect efforts to marstacimab-hncq, an injectable drug that prevents or reduces bleeding episodes in hemophilia A or B patients aged 12 years and older. LINK.

Sangamo Therapeutics to regain full rights to giroctocogene fitelparvovec hemophilia A gene therapy program as Pfizer exits
December 30, 2024 — Sangamo Therapeutics, Inc.—a genomic medicine company—announced it will regain full development and commercialization rights to giroctocogene fitelparvovec, an investigational gene therapy product candidate for the treatment of adults with moderately severe to severe hemophilia A that it has co-developed with, and licensed to Pfizer Inc., following a decision by Pfizer to terminate the global collaboration and license agreement between the parties. Sangamo intends to explore all options to advance the program, including seeking a potential new collaboration partner. LINK

SFDA approves concizumab-mtci for hemophilia A with inhibitors or hemophilia B with inhibitors
December 20, 2024 — The U.S. Food and Drug Administration (FDA) has approved concizumab-mtci as a once-daily prophylactic treatment to prevent or reduce the frequency of bleeding episodes for adults and children 12 years of age and older with hemophilia A or B with inhibitors. Alhemo is injected subcutaneously (under the skin) daily. LINK

Spark Therapeutics, a member of the Roche Group, introduces a new FVIII gene therapy variant while discontinuing the phase 3 dirloctocogene samoparvovec gene therapy study
December 6, 2024 — Spark Therapeutics has announced the decision to discontinue the phase 3 study of dirloctocogene samoparvovec gene therapy, while introducing an enhanced-function Factor VIII variant, aiming to develop a durable gene therapy product. Building on the capsid technology and scientific insights from the dirloctocogene samoparvovec program, Spark plans to initiate a phase 2b clinical study of the enhanced-function variant in the second half of 2025. The phase 3 dirloctocogene samoparvovec study was stopped before any participants were dosed, and the decision was not related to safety concerns with dirloctocogene samoparvovec.

European Commission approves marstacimab treatment for hemophilia A or B
November 20, 2024 — The European Commissionhas approved marstacimab—developed by Pfizer—for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and adolescent patients 12 years of age and older with hemophilia A without factor VIII inhibitors or hemophilia B without factor IX inhibitors (neutralizing antibodies). A notable benefit of marstacimab is that it is delivered subcutaneously once weekly through a pre-filled pen or syringe. LINK

Centessa Pharmaceuticals announces discontinuation of SerpinPC development
November 12, 2024 — Centessa Pharmaceuticals will discontinue the global clinical development of SerpinPC, an investigational serine protease inhibitor (SERPIN) engineered to specifically inhibit Activated Protein C (APC). SerpinPC was observed to have a favourable safety and tolerability profile; however, considering the evolving treatment and market landscape for hemophilia B, including the recent FDA approval of a similar product, Centessa decided to stop any further investment in this program. LINK

Spark Therapeutics pauses Phase 3 dirloctocogene samoparvovec gene therapy clinical study
October 16, 2024 — The following announcement was received by Spark: “After careful consideration, Spark Therapeutics, a member of the Roche Group, has elected to pause the Phase 3 dirloctocogene samoparvovec gene therapy clinical study while we evaluate the possibility to further transform outcomes for hemophilia A patients with an adeno-associated virus (AAV) gene therapy. This pause is not related to safety concerns with dirloctocogene samoparvovec. Spark will continue to work with investigators to ensure the necessary long-term follow-up with patients who have received dirloctocogene samoparvovec. Spark is dedicated to developing gene therapies that demonstrate safety, efficacy, durability, and predictability. Spark will provide an update as soon as they are able.”

FDA approves marstacimab-hncq treatment for hemophilia A or B
October 11, 2024 — The U.S. Food and Drug Administration (FDA) has approved marstacimab-hncq—developed by Pfizer—for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A without factor VIII inhibitors or hemophilia B without factor IX inhibitors (neutralizing antibodies). A notable benefit of marstacimab-hncq is that it is delivered subcutaneously once weekly through a pre-filled pen or syringe. LINK

Fidanacogene elaparvovec gene therapy approved for Europe
July 25, 2024 — The European Commission has approved fidanacogene elaparvovec, an adeno-associated viral (AAV) vector-based gene therapy for the treatment of hemophilia B in Europe. Developed by Pfizer, the treatment is for adults (aged 18 years or older) with moderately severe to severe hemophilia B (congenital Factor IX deficiency) who are negative for neutralizing antibodies to variant AAV serotype Rh74. LINK

NEJM publishes phase 3 data supporting the use of efanesoctocog alfa for treating children with severe hemophilia A
July 18, 2024 — Full results from the XTEND-Kids phase 3 study published in The New England Journal of Medicine (NEJM) highlights the efficacy, safety, and pharmacokinetic profile of Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein. Efanesoctocog alfa, a first-in-class, high-sustained factor VIII replacement therapy, is approved for adults and children with hemophilia A for routine prophylaxis and on-demand treatment to control bleeding episodes as well as for perioperative management (surgery). LINK

U.S. FDA approves gene therapy for hemophilia B
April 26, 2024 — Pfizer Inc. announced today that the U.S. Food and Drug Administration (FDA) has approved fidanacogene elaparvovec-dzkt for the treatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency) who:

  • Currently use factor IX prophylaxis therapy, or
  • Have current or historical life-threatening hemorrhage, or
  • Have repeated, serious spontaneous bleeding episodes, and,
  • Do not have neutralizing antibodies to the adeno-associated virus serotype used
LINK

Fidanacogene elaparvovec gene therapy approved for the treatment of hemophilia B
January 3, 2024 — Health Canada has approved fidanacogene elaparvovec, an adeno-associated viral (AAV) vector-based gene therapy for the treatment of hemophilia B which has been developed by Pfizer. The treatment is for adults (aged 18 years or older) with moderately severe to severe hemophilia B (congenital Factor IX deficiency) who are negative for neutralizing antibodies to variant AAV serotype Rh74. LINK

BioMarin Announces First Person Treated Commercially with valoctocogene roxaparvovec-rvox
August 30, 2023 — BioMarin Pharmaceutical Inc. announced today that an individual in Germany with severe hemophilia A was treated with valoctocogene roxaparvovec-rvox, marking the first time that this gene therapy has been given commercially in Europe. LINK

Roche discontinues hemophilia A gene therapy treatment candidate
July 28, 2023 — Roche is discontinuing the development of a mid-stage gene therapy candidate which was previously being assessed as a treatment for hemophilia A patients with inhibitors to factor VIII, the company announced Thursday. LINK

U.S. Food and Drug Administration Approves BioMarin’s valoctocogene roxaparvovec-rvox, the first gene therapy for adults with severe hemophilia A
June 29, 2023 — BioMarin today announced that the United States Food and Drug Administration (FDA) approved valoctocogene roxaparvovec-rvox gene therapy for the treatment of adults with severe hemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test. The one-time, single-dose infusion is the first approved gene therapy for severe hemophilia A in the U.S. LINK

CSL Behring Announces the first patient has received FDA-Approved etranacogene dezaparvovec-drlb for hemophilia B
June 20, 2023 — Etranacogene dezaparvovec-drlb (Hemgenix), the first FDA approved gene therapy for hemophilia B, has now been administered for the first time as an approved product to a person with hemophilia B. Hemgenix is also approved for use in the EU and has been granted conditional marketing authorization by the UK’s Medicines and Healthcare product Regulatory Agency. LINK

Two fitusiran Phase 3 studies published in The Lancet and The Lancet Haematology highlight potential to address unmet needs across all types of hemophilia.
April 4, 2023 — Both Phase 3 studies achieved their primary and secondary endpoints; fitusiran prophylaxis demonstrated significant and clinically meaningful improvements in bleed protection in people with hemophilia with and without inhibitors. LINK

BioMarin provides update on FDA review of valoctocogene roxaparvovec gene therapy for adults with severe hemophilia A
March 6, 2023 — BioMarin Pharmaceutical Inc. announced today that it received notice this afternoon from the U.S. Food and Drug Administration (FDA) that the agency has extended review of the company’s Biologics License Application (BLA) for valoctocogene roxaparvovec gene therapy for adults with severe hemophilia A. The FDA determined that the submission of the three-year data analysis from the ongoing Phase 3 GENEr8-1 study constituted a Major Amendment due to the substantial amount of additional data and set a new PDUFA Target Action Date of June 30, 2023. The company had previously communicated that this data submission could be qualified as a Major Amendment. LINK

Completed XTEND-Kids Phase 3 Sanofi and Sobi study strengthens potential of Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl for treatment of children <12 years of age with hemophilia A
March 2, 2023 — The XTEND-Kids phase 3 Sanofi and Sobi study evaluating the safety, efficacy and pharmacokinetics of Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl as once-weekly prophylaxis in previously treated patients <12 years of age with severe hemophilia A met its primary endpoint of safety, with no FVIII inhibitors detected in 74 children, with more than 50 children experiencing at least 50 exposure days, nearly a full year of treatment. The completion of XTEND-Kids represents the final milestone needed for regulatory submission in the EU. LINK

FDA approves once-weekly Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl a new class of factor VIII therapy for hemophilia A
February 23, 2023 – Sanofi announces that the U.S. Food and Drug Administration (FDA) has approved Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl, previously referred to as efanesoctocog alfa, a first-in-class, high-sustained factor VIII replacement therapy. This treatment is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as perioperative management (surgery) for adults and children with hemophilia A. LINK.

NEJM publishes once-weekly efanesoctocog alfa Phase 3 data for people with hemophilia A
January 25, 2023 – Pivotal study data published in The New England Journal of Medicine (NEJM) highlights the efficacy, safety, and pharmacokinetic profile of efanesoctocog alfa, an investigational treatment for hemophilia A. These data demonstrate that efanesoctocog alfa delivered normal to near-normal factor activity levels (>40%) for the majority of the week with once-weekly dosing. LINK

BioMarin Announces Stable and Durable Annualized Bleed Control at 3 years for ROCTAVIAN™
January 8, 2023 — BioMarin Pharmaceutical Inc. today announced positive results from three years of follow up from its ongoing global Phase 3 GENEr8-1 study of ROCTAVIANTM(valoctocogene roxaparvovec), an investigational one-time gene therapy for the treatment of adults with severe hemophilia A. This is the largest and longest global Phase 3 study to date for any gene therapy in hemophilia with 134 participants. LINK

Pfizer Announces Positive Top-Line Results from Phase 3 Study of Hemophilia B Gene Therapy Candidate
December 29, 2022 — Pfizer Inc. today announced positive top-line results from the Phase 3 BENEGENE-2 study evaluating fidanacogene elaparvovec, an investigational gene therapy, for the treatment of adult males with moderately severe to severe hemophilia B. LINK

EMA recommends granting a conditional marketing authorisation in EU for Hemgenix
December 16, 2022 — The European Medicines Agency (EMA) has recommended granting a conditional marketing authorisation in the European Union (EU) for CSL Behring GmbH’s Hemgenix (etranacogene dezaparvovec) for the treatment of severe and moderately severe haemophilia B in adults who do not have factor IX inhibitors (auto-antibodies produced by the immune system which make factor IX medicines less effective). Hemgenix is the first gene therapy to treat haemophilia B. It is delivered as a single infusion. LINK

CHMP recommends expansion of EU label for emicizumab to include people with moderate hemophilia A
December 16, 2022 — Roche announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended expansion of the emicizumab European Union (EU) marketing authorisation. If approved, the therapy would also be indicated for the routine prophylaxis of bleeding episodes in people with hemophilia A without factor VIII inhibitors who have moderate disease (FVIII ≥1% and ≤ 5%) with a severe bleeding phenotype. LINK.

U.S. Food and Drug Administration approves CSL’s HEMGENIX® (etranacogene dezaparvovec-drlb), the first gene therapy for hemophilia B
November 22, 2022 — CSL today announced that the U.S. Food and Drug Administration (FDA) approved HEMGENIX® (etranacogene dezaparvovec-drlb), the first one-time gene therapy for appropriate adults with hemophilia B. HEMGENIX is approved for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage or have repeated, serious spontaneous bleeding episodes. LINK

Freeline takes difficult decision to not progress their hemophilia B program into Phase 3 development
November 15, 2022 — As a result of a previously announced evaluation of its investigational gene therapy program for hemophilia B, Freeline has decided to focus its resources on other programs, and stop investment in further development of it’s hemophilia B program without a partner. LINK

First Gene Therapy for Adults with Severe Hemophilia A, BioMarin’s ROCTAVIAN (valoctocogene roxaparvovec), Approved by European Commission (EC) SAN RAFAEL, Calif.
Aug. 24, 2022 — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the European Commission (EC) has granted conditional marketing authorization (CMA) to ROCTAVIAN (valoctocogene roxaparvovec) gene therapy for the treatment of severe hemophilia A (congenital Factor VIII deficiency) in adult patients without a history of Factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5). LINK

Monkeypox Virus and Plasma Protein Therapies
27 June 2022 — PPTA considers that the current MPXV outbreak is not a concern for the safety margins of plasma protein therapies manufactured by PPTA member companies. This assessment is also shared by other concerned parties (AABB, WHO). PDF

Precision BioLogic Launches New Product to Improve Testing for Hemophilia B
MONTREAL – May 9, 2022 /CNW/ — Precision BioLogic Inc., a leading developer of hemostasis diagnostic products, is pleased to announce the launch of its new CRYOcheck Chromogenic Factor IX assay in Canada, the European Union, Australia, and New Zealand following market authorization by Health Canada and respective in-country regulatory authorities. The announcement was made at the World Federation of Hemophilia Congress taking place in Montreal, May 8–11, 2022. LINK

European Medicines Agency Commences Review of Novel Gene Therapy Candidate Etranacogene Dezaparvovec for People with Hemophilia B
MARBURG, GERMANY – March 28, 2022 — Global biotherapeutics leader CSL Behring today announced that the European Medicines Agency (EMA) has accepted the Marketing Authorization Application (MAA) for etranacogene dezaparvovec (EtranaDez) under its accelerated assessment procedure. LINK

BioMarin Announces Publication in New England Journal of Medicine of One-Year Results from Phase 3 Pivotal Trial with Valoctocogene Roxaparvovec Gene Therapy in Adults with Severe Hemophilia A
SAN RAFAEL, Calif. – Mar. 17, 2022 /PRNewswire/, Ozelo, M. et al., Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A. N Engl J Med 2022; 386:1013-1025, DOI: 10.1056/NEJMoa2113708 — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced publication of results from the Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A, in the New England Journal of Medicine (NEJM). LINK

Biomarin gives an update and reports a serious adverse event for its Phase 3 hemophilia A gene therapy study
Feb. 4, 2022 — An update for the hemophilia community from BioMarin, regarding the ongoing Phase 3 BioMarin hemophilia A gene therapy study, and a serious adverse event deemed, by the Independent Data Safety Monitoring Committee, unrelated to the investigational gene therapy from the phase 1/2 study. PDF

BioMarin Announces Stable and Durable Annualized Bleed Control in the Largest Phase 3 Gene Therapy Study in Adults with Severe Hemophilia A
SAN RAFAEL, Calif. – Jan. 9, 2022 — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced positive results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. This is the largest global Phase 3 study to date for any gene therapy in hemophilia, with 134 participants. LINK

Sigilon Therapeutics Announces Strategic Reprioritization
CAMBRIDGE, Mass. – Dec. 13, 2021 (GLOBE NEWSWIRE) — Sigilon Therapeutics, Inc. (NASDAQ: SGTX), a biotechnology company that seeks to develop functional cures for chronic diseases through its Shielded Living Therapeutics platform, today announced a strategic reprioritization to enable the Company to focus on MPS-1 and diabetes. PDF

Sigilon Therapeutics Announces Update on SIG-001 Phase 1/2 Study in Hemophilia A
CAMBRIDGE, Mass. – Nov. 29, 2021 (GLOBE NEWSWIRE) — Sigilon Therapeutics, Inc. (NASDAQ:SGTX), a biotechnology company that seeks to develop functional cures for chronic diseases through its Shielded Living Therapeutics platform, today reported that fibrosed spheres were observed during a retrieval procedure in a patient in its Phase 1/2 study of SIG-001 in severe or moderately severe hemophilia A. PDF

FDA places the Pfizer/Sangamo Therapeutics phase 3 AFFINE haemophilia A gene therapy study on clinical hold
November 5, 2021 — On Tuesday, November 4, the U.S. Food and Drug Administration (FDA) placed the Pfizer/Sangamo haemophilia A gene therapy programme, including the pivotal phase 3 AFFINE study ( NCT04370054 ), with giroctocogene fitelparvovec (SB-525 or PF-07055480), on clinical hold until the review of a proposed protocol amendment. ( Sangamo news release ). LINK

Spark Therapeutics’ SPK-8011 Suggests Stable and Durable Factor VIII Expression in Largest Phase 1/2 Gene Therapy Study in Hemophilia A to Date
PHILADELPHIA – July 21, 2021 — Spark Therapeutics, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) and a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, today announced updated data from the ongoing Phase 1/2 clinical trial of investigational SPK-8011 in hemophilia A during the International Society of Thrombosis and Hemostasis (ISTH) 2021 Virtual Congress (July 17-21). LINK

European Medicines Agency Validates BioMarin's Marketing Authorization Application for Valoctocogene Roxaparvovec to Treat Severe Hemophilia A
SAN RAFAEL, Calif. – July 15, 2021 (PRNewswire) — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the European Medicines Agency (EMA) validated the Company's Marketing Authorization Application (MAA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. With today's validation the MAA review can now commence. A CHMP opinion is anticipated in the first half of 2022. LINK

uniQure Announces Positive 52-Week Clinical Data from HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B and Provides Regulatory Update
LEXINGTON, Mass. and AMSTERDAM, The Netherlands – June 22, 2021 (GLOBE NEWSWIRE) — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced positive 52-week clinical data on all patients from its pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec, an investigational adeno-associated virus five (AAV5)-based gene therapy for the treatment of patients with severe and moderately severe hemophilia B. These are the first clinical data to be reported from a Phase III gene therapy study in hemophilia B and, with 54 patients, the largest set of hemophilia B patients receiving a single gene therapy investigational product to date. LINK

European Medicines Agency Grants BioMarin's Request for Accelerated Assessment of Valoctocogene Roxaparvovec for Treatment of Severe Hemophilia A
SAN RAFAEL, Calif. – May 24, 2021 (PRNewswire) — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the European Medicines Agency (EMA) has granted its request for accelerated assessment of valoctocogene roxaparvovec, for adults with severe hemophilia A. Accelerated assessment reduces the time frame for the EMA Committee for Medicinal Products for Human Use (CHMP) and Committee for Advanced Therapies (CAT) to review a MAA for an Advanced Therapy Medicinal Product (ATMP). A CHMP opinion is expected in the first half of 2022. LINK

CSL Behring Announces Closing of Global Commercialization and License Agreement with uniQure
KING OF PRUSSIA, PA, USA – May 6, 2021 — Global biotherapeutics leader CSL Behring today announced the closing of its global Commercialization and License agreement with uniQure (NASDAQ: QURE) for etranacogene dezaparvovec (AMT-061), a novel gene therapy for the treatment of hemophilia B. CSL Behring has been recognized as a global hematology leader for decades. The acquisition complements both CSL Behring’s cell and gene therapy scientific platform and its hematology product portfolio, which include other treatments for hemophilia B as well as therapies for treating hemophilia A, von Willebrand disease, thrombosis, and other life-threatening conditions. LINK

uniQure announces closing of commercialization and license agreement with CSL Behring
LEXINGTON, Mass. and AMSTERDAM, The Netherlands – May 06, 2021 (GLOBE NEWSWIRE) — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 for the global licensing agreement with CSL Behring for etranacogene dezaparvovec, an investigational gene therapy for patients with hemophilia B, expired on May 5, 2021, and the agreement became fully effective today, May 6, 2021. The agreement was contingent on completion of review under antitrust laws in the United States, Australia, and the United Kingdom. The antitrust review process is now complete in all three countries. PDF

uniQure Announces FDA Removes Clinical Hold on Hemophilia B Gene Therapy Program
LEXINGTON, Mass. and AMSTERDAM, The Netherlands – April 26, 2021 (GLOBE NEWSWIRE) — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced today that the U.S. Food and Drug Administration (FDA) has removed the clinical hold on the Company’s hemophilia B gene therapy program after determining the Company satisfactorily addressed all issues identified by the FDA related to a single patient diagnosed with hepatocellular carcinoma (HCC) in the HOPE-B pivotal trial. PDF

uniQure Announces Findings from Reported Case of Hepatocellular Carcinoma (HCC) in Hemophilia B Gene Therapy Program
LEXINGTON, Mass. and AMSTERDAM, The Netherlands – March 29, 2021 (GLOBE NEWSWIRE) — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced today the results of a comprehensive investigation into the case of hepatocellular carcinoma (HCC) diagnosed in one patient in the HOPE-B pivotal trial of etranacogene dezaparvovec. The results of the investigation found that it is highly unlikely the HCC was caused by etranacogene dezaparvovec. LINK

Takeda Announces Strategic Partnership to Accelerate the Development of Enzyre’s Diagnostic Technology Platform That Aims to Improve the Standard of Care for Patients with Bleeding Disorders
OSAKA, JAPAN – March 4, 2021 — Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE:TAK) (“Takeda”) today announced it has entered into a strategic partnership with Enzyre to accelerate the development of Enzyre’s proprietary platform Enzypad to enable patients to test their blood coagulation in a home setting. The partnership builds on an existing research collaboration agreement with Enzyre signed in December 2019 and close collaboration over the last five years. LINK

Data at EAHAD 2021 Highlight Value of Takeda’s Leading Hematology Portfolio in Clinical Settings and Commitment to Patient-Focused Advancements
OSAKA, JAPAN – February 3, 2021 — Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE:TAK) (“Takeda”) today announced that 14 abstracts from the company’s Hematology portfolio and pipeline are being presented as electronic posters at the Virtual European Association for Haemophilia and Allied Disorders Congress (EAHAD 2021). Data include multiple abstracts reinforcing the long-term efficacy and safety of Takeda’s hematology portfolio in clinical practice, supporting the value of these treatments in a real world setting. PDF

BioMarin Announces Positive Phase 3 Gene Therapy Trial Results in Adults with Severe Hemophilia A; Study Met All Primary and Secondary Efficacy Endpoints in One-Year Data Set
SAN RAFAEL, U.S.A. – January 10, 2021 — BioMarin Pharmaceutical Inc. today announced positive topline results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. LINK

UniQure Reports Development of Hepatocellular Carcinoma in a Gene Therapy Clinical Trial Participant
Lexington, MA and Amsterdam, the Netherlands – December 21, 2020 (BUSINESS WIRE) — UniQure, which is running a Phase 3 AAV5-Factor IX gene therapy study, reported today a case of hepatocellular carcinoma (HCC) in a trial participant1. HCC is a tumor that arises in the liver. LINK

Pfizer Doses First Participant in Phase 3 Study Evaluating anti-TFPI Investigational Therapy, Marstacimab, for People With Severe Hemophilia A and B With or Without Inhibitors
NEW YORK, U.S.A. – November 23, 2020 (BUSINESS WIRE) — Pfizer Inc. (NYSE: PFE) today announced that the first participant has been dosed in the Phase 3 BASIS study of marstacimab (PF-06741086), an anti-tissue factor pathway inhibitor (anti-TFPI) being evaluated for the treatment of people with severe hemophilia A or B, with or without inhibitors. LINK

uniQure Announces Positive Top-Line Data from the HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B
LEXINGTON, Ma. – November 19, 2020 (GLOBE NEWSWIRE) — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced positive top-line data from its pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec, an investigational adeno-associated virus five (AAV5)-based gene therapy for the treatment of patients with severe and moderately severe hemophilia B. This is the first data set to be reported from a Phase III gene therapy study in hemophilia B and, with 54 patients, the largest set of patients receiving a single gene therapy investigational product to be reported to date. These clinical data were published today as a late-breaking abstract, one of only six accepted for presentation at the 62nd Annual Meeting of the American Society of Hematology (ASH) and will be featured as an oral presentation in the conference on December 8, 2020. LINK.

Takeda to Present Rare Bleeding Disorders Research at Thrombosis & Hemostasis Summit of North America (THSNA) 2020
CAMBRIDGE, Ma. and OSAKA, JAPAN – October 27, 2020 — Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE:TAK) (“Takeda”), today presented four abstracts at the Thrombosis and Hemostasis Summit of North America (THSNA), highlighting Takeda’s commitment to advancing personalized care in people living with bleeding disorders. LINK

rHEALTH’s FVIII Point-of-Care Device Awarded $1.5M Direct-to- Phase II Grant from the NIH
BEDFORD, Ma. – October 8, 2020 — rHEALTH LLC, a Diagnose Yourself, Anywhere™ company, today announced that its technology is the subject of the award from the National Institute of Health (NIH). The award is to rHEALTH’s affiliate, the DNA Medicine Institute (DMI), which has granted rHEALTH commercial rights to the technology. The focus of the grant is to further develop the company’s fingerstick-based point-of-care technology for monitoring both FVIII and emicizumab. PDF

An update from Roche on the evolution of Hemlibra post-approval data communications
September 23, 2020 — At Roche we recognise and appreciate the interest that the haemophilia community has in regards to Hemlibra (emicizumab), specifically around the subject of its long-term efficacy and safety, both now and in the future. We understand that the subject of long-term safety for haemophilia treatments is an important topic also for the community, and at Roche we will continue to listen to the needs of the community and aspire to provide information that leads to better clinical decision making and patient care. PDF

New England Journal of Medicine publishes positive final results from phase 1/2a study of BIVV001 in people with severe haemophilia A
Stockholm & Paris – September 10, 2020 — The New England Journal of Medicine today published positive final results from the phase 1/2a trial evaluating the safety, tolerability and pharmacokinetics of BIVV001 (rFVIIIFc-VWF-XTEN) in adult patients with severe haemophilia A. BIVV001 is an investigational factor VIII therapy designed to provide higher bleed protection in a once-weekly prophylactic treatment regimen. Sobi (STO:SOBI) and Sanofi collaborate on the development and commercialization of BIVV001. LINK

BioMarin Receives Complete Response Letter (CRL) from FDA for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A
SAN RAFAEL, Calif. – August 19, 2020 — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) to the Company's Biologics License Application (BLA) for valoctocogene roxaparvovec gene therapy for severe hemophilia A on August 18, 2020. The FDA issues a CRL to indicate that the review cycle for an application is complete and that the application is not ready for approval in its present form. LINK

Precision BioLogic Inc. announces CRYOcheck™ Chromogenic Factor VIII Assay Cleared for Sale in U.S
HALIFAX – July 20, 2020 — Precision BioLogic Inc., a leading developer of hemostasis diagnostic products, is pleased to announce FDA 510(k) clearance and the launch of its new CRYOcheck Chromogenic Factor VIII assay in the U.S. This clearance follows authorizations received from regulatory authorities in Canada, the European Union, Australia, and New Zealand where the assay launched earlier this year. PDF

Takeda Provides Updates on Its Pharmacokinetic-guided Prophylaxis Studies at ISTH 2020, Reinforcing Its Commitment to Advancing Personalized Care for People with Bleeding Disorders
OSAKA, JAPAN – July 12, 2020 — Takeda Pharmaceutical Company Limited (“Takeda”) (TSE: 4502/NYSE:TAK) today announced that 13 abstracts are being presented from the company’s Hematology portfolio and pipeline at the International Society on Thrombosis and Haemostasis 2020 Virtual Congress (ISTH 2020). Among the data, Takeda spotlighted four abstracts to highlight its commitment to advancing personalized care through pharmacokinetic (PK)-guided prophylaxis in people living with hemophilia or von Willebrand Disease (VWD) – including scientific updates in patients with hemophilia A from the Phase 3 PROPEL and Phase 3b CONTINUATION studies investigating potential benefits of personalized TAK-660 (rurioctocog alfa pegol) prophylaxis. Two population studies into the PK/pharmacodynamic (PD) profiles of recombinant von Willebrand factor (rVWF), which provide data to assist in the optimization of rVWF personalized dosing strategies, were also presented. LINK

Spark Therapeutics Announces Updated Data on SPK-8011 from Phase 1/2 Clinical Trial in Hemophilia A at ISTH 2020 Virtual Congress
PHILADELPHIA, Pa. – July 12, 2020 — Spark Therapeutics, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) and a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, today announced updated data from three dose cohorts of the ongoing Phase 1/2 clinical trial of investigational SPK-8011 in hemophilia A. These data were presented at the International Society of Thrombosis and Hemostasis (ISTH) 2020 Virtual Congress by Principal Investigator Lindsey A. George, M.D., The Perelman School of Medicine, University of Pennsylvania and Children’s Hospital of Philadelphia. LINK

CSL Behring Agrees to Acquire Novel Late-Stage Gene Therapy Candidate for Hemophilia B Patients from uniQure
KING OF PRUSSIA, Pa. – June 24, 2020 — Global biotherapeutics leader CSL Behring announced today that it has agreed to acquire exclusive global license rights to commercialize an adeno-associated virus (AAV) gene therapy program, AMT-061 (etranacogene dezaparvovec), for the treatment of hemophilia B from uniQure (NASDAQ: QURE), a leading gene therapy company. The AMT-061 program, currently in Phase 3 clinical trials, could be one of the first gene therapies to provide potentially long-term benefits to patients with hemophilia B. LINK

BioMarin Provides Highlights of 4 Years of Clinical Data from Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A
SAN RAFAEL, Calif. – May 31, 2020 — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today an update to its previously reported results of an open-label Phase 1/2 study of valoctocogene roxaparvovec, an investigational gene therapy treatment for adults with severe hemophilia A. The data have been submitted as a late-breaking abstract to the World Federation of Hemophilia (WFH) Virtual Summit to be held June 14-19, 2020. LINK

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Eventos próximos

Hoy

  • Webinario de la FMH: Aspectos destacados de la tercera edición del Manual de laboratorio 2025

    Únete a nosotros para nuestra próxima serie de seminarios web sobre el Manual de Laboratorio que comenzará el jueves 2 de octubre de 2025, a las 3:00 p.m. UTC. Los seminarios web se llevarán a cabo en inglés con interpretación en vivo al español. Esta serie de tres partes continuará el 6 de noviembre y el 4 de diciembre de 2025, proporcionando información práctica y discusiones para profesionales de laboratorio.

  • Webinario de la FMH: Comprehensive Pain Management in People with Bleeding Disorders (PWBD)

    Join the WFH MSK Committee webinar “Comprehensive Pain Management in People with Bleeding Disorders (PWBD)” on December 12, 2025, from 9:00–10:30 a.m. (Montreal time). Experts will discuss the causes of pain in PWBD, pharmacological treatments, and the role of strength training in managing arthropathy. This session is designed for healthcare professionals seeking practical, multidisciplinary strategies to improve pain management and overall care for people with bleeding disorders.

  • Día mundial de la hemofilia 2026

    El 17 de abril de 2026, la comunidad mundial de trastornos de la coagulación hereditarios se reunirá con motivo del Día mundial de la hemofilia para abogar por todos los trastornos de la coagulación hereditarios. El tema de este año, El diagnóstico: primera etapa de la atención, destaca la importancia crucial del diagnóstico, el primer paso indispensable para el tratamiento y la atención.

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Disclaimer

The information on the WFH website is provided for general information purposes only. The WFH does not engage in the practice of medicine and under no circumstances recommends particular treatment for specific individuals. For diagnosis or consultation on a specific medical problem, the WFH recommends that you contact your physician or local treatment centre. Before administering any products, the WFH urges patients to check dosages with a physician or hemophilia centre staff, and to consult the pharmaceutical company’s printed instructions.

While every effort has been made to ensure the accuracy of the information on this site, the WFH does not guarantee the information is accurate, and is not responsible in any way whatsoever for damages arising out of the use of this website or any of the information contained herein.

Messages posted to WFH discussion forums, Facebook, Twitter, and other social media platforms do not represent the opinions of the World Federation of Hemophilia, its staff, or Board of Directors. The author of a message is solely responsibility for its content. Information posted on WFH social networks and platforms should never be a substitute for individualized professional medical advice, even when the author has medical qualifications or is considered an authority. Information posted to a discussion group should not be used to diagnose or treat a specific health problem without consulting a qualified healthcare professional. The WFH recommends that you contact your physician or local treatment centre if you have any individual questions or concerns.

References and links to other websites or references to other organizations, products, services, or publications do not constitute endorsement or approval by the WFH. The WFH is not responsible and assumes no liability for the content of any linked websites.

Fraud Alert

Unauthorized solicitations – Warning

The WFH has been made aware of various correspondences—circulated via e-mail and telephone—coming from individuals impersonating WFH staff or falsely stating that they are associated with the WFH. These correspondences, which may seek to obtain money using the name of someone affiliated with the WFH, are fraudulent and come from outside of our organization.

If you receive a suspicious solicitation, exercise extreme caution. In the case of an email, look at the email address to see if it looks suspicious (for example, all WFH emails come from @wfh.org).

We are asking you to remain vigilant, and if you have any doubts about the correspondence, please forward the email to the WFH at [email protected] or call +1 514-875-7944.

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Hemophilia Alliance

Recognizing that training women leaders ensures the diversity our community need to thrive; the Hemophilia Alliance has been a longtime supporter of the Susan Skinner Memorial Fund. They deepened their engagement with the global community through the establishment of the Travel Grant Program, which allows US-based multidisciplinary healthcare professionals the opportunity to attend WFH global training workshops. The wealth of experience and perspective attendees bring back home to their communities helps patients across the country. Their patience and support as we grappled with changing norms in these challenging times gave us the flexibility to meet our community’s needs with revamped programming.

Hemophilia of Georgia

Hemophilia of Georgia has stood with us for over three decades, playing an early role in distributing donated factor via the WFH Humanitarian Aid program. Our relationship has deepened over the years as they continued to support this program and came on board as a founding Chapter Challenge champion in 2016. Their belief in educating future leaders is clear in their investment in the Youth Leadership Workshops, which has led to the training of over 50 young adults. They also support workshops devoted to educating providers and patient leaders on how to collect and use data to advocate for better patient care. Their staff generously volunteer their time as trainers and as well as serving on various committees.

National Bleeding Disorders Foundation

The National Bleeding Disorders Foundation helps advance our mission on several fronts. They inspire others to follow their lead as a founding champion of the NHF Chapter Challenge. Their commitment to equality of access is demonstrated in their support of the Cornerstone Initiative, which lays the foundation for care in the world's most underserved regions, as well as programs that aim to raise awareness and improve care of women with bleeding disorders and those with von Willebrand disease. We also benefit from their steadfast engagement with various programs including the WFH Twinning Program and their individual leaders’ contribution to our leadership and committees.

Legacy Gift

Do you sometimes wish you could do more? A bequest is a simple way for you to make a significant gift without changing anything about your financial security and lifestyle today. Your legacy will sustain care for the generations to come.

Download these sample clauses to give to your legal advisor.

We understand if you prefer to remain anonymous, but we would be very honoured if you would let us know of your intentions so that we may thank you personally. We would also be happy to answer any questions you may have or give you additional information. Please contact Jennifer Laliberté at 514‑875‑7944 ext.2822 or [email protected].

Disclaimer

The information on the WFH USA website is provided for general information purposes only. The WFH USA does not engage in the practice of medicine and under no circumstances recommends particular treatment for specific individuals. For diagnosis or consultation on a specific medical problem, the WFH USA recommends that you contact your physician or local treatment centre. Before administering any products, the WFH USA urges patients to check dosages with a physician or hemophilia centre staff, and to consult the pharmaceutical company’s printed instructions.

While every effort has been made to ensure the accuracy of the information on this site, the WFH USA does not guarantee the information is accurate, and is not responsible in any way whatsoever for damages arising out of the use of this website or any of the information contained herein.

Messages posted to WFH USA discussion forums, Facebook, Twitter, and other social media platforms do not represent the opinions of the World Federation of Hemophilia, its staff, or Board of Directors. The author of a message is solely responsibility for its content. Information posted on WFH USA social networks and platforms should never be a substitute for individualized professional medical advice, even when the author has medical qualifications or is considered an authority. Information posted to a discussion group should not be used to diagnose or treat a specific health problem without consulting a qualified healthcare professional. The WFH USA recommends that you contact your physician or local treatment centre if you have any individual questions or concerns.

References and links to other websites or references to other organizations, products, services, or publications do not constitute endorsement or approval by the WFH USA. The WFH USA is not responsible and assumes no liability for the content of any linked websites.

Website privacy policy

The WFH USA does not collect personally identifying information about you when you visit our site, unless you choose to provide such information to us. Providing such information is strictly voluntary. This policy is your guide to how we will handle information we learn about you from your visit to our website.

Use of links

Throughout our website, we provide links to other servers which may contain information of interest to our readers. We take no responsibility for, and exercise no control over, the organizations, views, or accuracy of the information contained on other servers. When linking to the WFH USA, we request that you ensure that there are no associated connections for commercial purposes. Any official use of the name WFH USA or the use of its logo needs to be approved by the WFH USA. If you have a link you’d like us to consider adding to our website, please send an email to [email protected] with the subject “Link request.”

Use of text and images

All information, photographs, graphics, and other material on this website are protected by copyright. While users of this website may use the content for personal, non-commercial use, none of the content of this website can be modified, translated, transferred or sold, copied, reproduced, published, or distributed, in whole or in part, without the prior written consent of the WFH USA. To view a copy of the WFH USA’s permissions guidelines, click here.

The WFH USA encourages the dissemination of its information and welcomes proposals on the reprinting, redistribution, and translation of its materials. If you would like to publish information that you find on our website, please send your request to [email protected].

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If you have suggestions on how to make the site more accessible, please contact us at [email protected].

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Our website provides users the opportunity to opt-out of receiving communications from us through a special online form or via the communication they have received. You may, at all times, choose to receive which communications within the organization you can receive or none at all. You may also update your contact information previously provided to us via another online form.

Questions about our policies

If you have any questions about this privacy statement, the practices of this site, or your dealings with this website, you can contact us at [email protected].

Fraud alert

Unauthorized solicitations – Warning

The WFH USA has been made aware of various correspondences—circulated via e-mail and telephone—coming from individuals impersonating WFH USA staff or falsely stating that they are associated with the WFH USA. These correspondences, which may seek to obtain money using the name of someone affiliated with the WFH USA, are fraudulent and come from outside of our organization.

If you receive a suspicious solicitation, exercise extreme caution. In the case of an email, look at the email address to see if it looks suspicious (for example, all WFH USA emails come from @wfhusa.org or @wfh.org).

We are asking you to remain vigilant, and if you have any doubts about the correspondence, please forward the email to the WFH USA at [email protected] or call 1-877-417-7944.

Premio internacional de la FMH por servicios destacados - receptores

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outstanding-service-award-2010

Premio internacional de la FMH al voluntariado por la atención de la salud - receptores

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healthcare-award-2018
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AÑOS PASADOS

2012 Piet de Kleijn
Países Bajos
2010 Dr. Mammen Chandy
India
2008 Dr. Man-Chiu Poon
Canadá
2006 Dr. Norma de Bosch
Venezuela
2004 Dr. Parttraporn Insarangkura
Tailandia

Dr. Carol K. Kasper
Estados Unidos

Premios a los hermanamientos del año

2022 Ammán (Jordania) – Dublín (Irlanda)
2021 Addis Abeba (Etiopía) – Minneapolis (EE.UU.) y
Teherán (Irán) – Milán (Italia)
2020 Aluva (India) – Newcastle (Reino Unido)
2019 Yogyakarta / Solo (Indonesia) – Utrecht (Países Bajos)
2018 Abidjan (Costa de Marfil) – Bruselas (Bélgica)
2017 Santa Cruz de la Sierra (Bolivia) – Buenos Aires (Argentina)
2016 Kampala (Uganda) – Londres (Reino Unido)
2015 Varna (Bulgaria) – Bonn (Alemania)
2014 Arequipa (Perú) – Los Ángeles (Estados Unidos)
2013 Davangere (India) – Liverpool (Reino Unido)
Ludhiana (India) – Detroit (Estados Unidos)
2012 Hanói (Vietnam) – Melbourne (Australia)
2011 Manado (Indonesia) – Utrecht (Países Bajos)
2010 Delhi (India) – Winnipeg (Canadá)
Yaoundé (Camerún) – Ginebra (Suiza)
2009 Chisinau (Moldovia) – Varsovia (Polonia)
Colombo (Sri Lanka) – Vellore (India)
2008 Gaborone (Botswana) – Filadelfia (Estados Unidos)
2007 Vientiane (Laos) – Brest (Francia)
Damasco (Siria) – Montpellier (Francia)
2006 Lima (Perú) – Fort Worth (Estados Unidos)
2005 Ereván (Armenia) – Minneapolis (Estados Unidos)
Casablanca (Marruecos) – Caen (Francia)
2004 Beirut (Líbano) – Ginebra (Suiza)
Santo Domingo (República Dominicana) – Caracas (Venezuela)
2003 El Cairo (Egipto) – Knoxville (Estados Unidos)
2002 Moscú (Rusia) – Liverpool (Reino Unido)
Panamá – Valencia (España)
2001 Bangalore (India) – St. Louis (Estados Unidos)
2000 Pune (India) – Bradford (Reino Unido)
1999 Montevideo (Uruguay) – Buenos Aires (Argentina)
Tianjin (China) – Calgary (Canadá)
1998 Plovdiv (Bulgaria) – Bonn (Alemania)
1997 Bogotá (Colombia) – Los Ángeles (Estados Unidos)
Tartu (Estonia) – Estocolmo (Suecia)
1996 Timisoara (Rumanía) – Múnich (Alemania)
Riga (Letonia) – Munster (Alemania)
1995 Klaipeda (Lituania) – Malmo (Suecia)
1994 Bratislava (Eslovaquia) – Tel Hashomer (Israel)

Premio Pietrogrande

2025 Greig Blamey
Canadá
2023 Nicholas Goddard
Reino Unido
2021 Axel Seuser
Alemania
2019 Dr. Adolfo Llinás
Colombia
2017 Pamela Hilliard
Canadá
2015 Kathy Mulder
Canadá
2013 James Luck
Estados Unidos
2012 Piet de Kleijn
Países Bajos
2011 Dr. Lily Heijnen
Países Bajos
2010 Dr. Horacio Caviglia
Argentina
2009 Jerome D. Wiedel
Estados Unidos
2008 Dr. Federico Fernández-Palazzi
Venezuela
2007 Michael Heim
Israel
2006 Brenda Buzzard
Reino Unido
2005 Dr. Marvin Gilbert
Estados Unidos

Premio Henri Horoszowski

2025 Shubhranshu S. Mohanty
India		 Haemophilic pseudotumour – New improvised classification system and its outcome in 168 cases at a Tertiary care centre
2021 Baolai Hua
China		 The effects of intra-articular injection of TNF-alpha Antagonists in treatment of haemophilic synovitis
2019 Maarten Eerdekens
Bélgica		 Asociación entre la cinética multisegmento del pie y el daño articular inducido por la sangre en pacientes adultos con artropatía hemofílica del tobillo
2017 Fabio Souza
Brasil		 Decrease in joint inflammation, swelling and pain after a swimming protocol in animal model of experimental-induced hemarthrosis
2015 SM Javd Mortazavi
Irán		 Bilateral total knee arthroplasty in patients with hemophilia: A safe and cost-effective procedure
2013 Lize F.D. van Vulpen
Estados Unidos		 Una proteína de fusión de IL4 e IL10 es igualmente eficaz para proteger el cartílago del daño inducido por la sangre en comparación con los componentes
2012 Horacio Caviglia
Argentina		 Cirugía de pseudotumor en pacientes con hemofilia A: resultados comparativos entre pacientes inhibidores y no inhibidores
2011 Nick Goddard
Reino Unido y
Monique van Meegeren
Países Bajos		 La administración temprana de IL-4 e IL-10 previene el daño del cartílago inducido por la sangre in vitro
2010 Lydia Abad-Franch
España		 Influencia de un protocolo de entrenamiento acuático en la reducción del riesgo de morbilidad y mortalidad cardiovascular en un hemofílico
2009 Sebastien Lobet
Bélgica		 Impacto de la artropatía del tobillo con hemofilia en la discapacidad de la marcha: análisis de variables energéticas y mecánicas
2008 Jose Alberto Tlacuilo-Parra
 México		 Marcadores de rotación ósea y densidad mineral ósea en niños con hemofilia
2007 Natalie Jansen
Países Bajos		 Capacidades protectoras de la interleucina-10 en el daño del cartílago inducido por la sangre
2006 Axel Seuser y E. Kusch
Alemania		 Estudio multicéntrico del resultado ortopédico de las extremidades inferiores en 249 niños con hemofilia
2005 Frank van Genderen, Nico van Meeteren, Lily Heijnen, Piet de Kleijn, Marijke van den Berg, Paul Helders
Países Bajos		 Lista de actividades para la hemofilia: desarrollo final y validación de un cuestionario de autoevaluación específico para hemofilia sobre las capacidades funcionales
2004 Goris Roosendaal, Zalima N Jahangier, Kim MG Jacobs, Johannes WJ Bijlsma, Floris PJG Lafeber
Países Bajos		 La radiosinoviortesis con YTTRIUM-90 da como resultado efectos directos adversos sobre el cartílago: un estudio in vitro en humanos
2003 Axel Seuser, Thomas Wallny, Günter Schumpe, Elmar Trunz, Hans-Hermann Brackman
Alemania		 ¿Cómo aconsejar a los jóvenes hemofílicos que encuentren el deporte adecuado? Un algoritmo nuevo y seguro
2002 James Luck, Jr. y Mauricio Silva
Estados Unidos		 Revisión a largo plazo de la artroplastia total de rodilla
2001 Federico Fernández-Palazzi
Venezuela		 Clorhidrato de oxitetraciclina: un nuevo material para las órtesis sinoviales químicas
2000 Horacio Caviglia
Argentina		 Pseudotumores
1999 William J. Ribbans y J.L. Hicks
Reino Unido		 Comunicación y cooperación para el nuevo milenio: el futuro del comité musculoesquelético en el siglo XXI
1998 Adolfo Llinas, Mauricio Silva, Monica Duarte, Gonzalo Ucros, Graciela Perini, Angela Maria Cerquera, Andres Forero
Colombia		 La sinoviortesis de fosfato crómico sin restricción de edad debería ser la primera opción de tratamiento para la sinovitis hemofílica activa crónica

Escalas funcionales

Los resultados de interés recolectados en el RMTC abarcan características demográficas, resultados médicos y relacionados con el tratamiento, resultados de la carga de la enfermedad, y resultados reportados por los pacientes.

El RMTC incluye cinco escalas funcionales (en inglés) como parte de su conjunto de datos ampliados:

Conjuntos de datos de hemofilia del RMTC

Conjunto de datos mínimo del RMTC

El conjunto de datos mínimo (CDM) es un grupo de datos demográficos, sobre diagnóstico, síntomas y tratamiento que se recolecta de los pacientes que participan en el RMTC.

Conjunto de datos ampliado del RMTC

El conjunto de datos ampliado (CDA)es un grupo de datos más grande, con mayores detalles que el CDM, que abarca preguntas sobre datos demográficos, diagnóstico, síntomas, tratamiento y carga de la enfermedad de los pacientes. Los centros de tratamiento de hemofilia pueden utilizar el CDA para recolectar información de pacientes que participan en el RMTC. El conjunto de datos ampliado (CDA) del RMTC se agregó a la base de datos en marzo del 2019.

Estados Unidos - Columbus, Ohio

Año de designación del CIEHEMO: 2018

Nationwide Children’s Hospital

Áreas de especialidad

  • Cuidados dentales
  • Diagnóstico de laboratorio
  • Enfermería
  • Cirugía y cuidados ortopédicos
  • Servicios de hematología pediátrica
  • Fisioterapia/Terapia ocupacional
  • Psicología/Servicios sociales

Estados Unidos - Houston, Texas

Año de designación del CIEHEMO: 2000

Gulf States Hemophilia and Thrombophilia Center
University of Texas Health Science Center at Houston

Áreas de especialidad

  • Hematología pediátrica y adultos
  • Diagnóstico de laboratorio y hemostasia
  • Enfermería
  • Banco de sangre
  • Fisioterapia/Terapia ocupacional
  • Psicología/Servicios sociales

Estados Unidos - Los Ángeles, California

Año de designación del CIEHEMO: 1970

Lifespan Orthopaedic Hemophilia Treatment Center at Luskin Orthopedic Institute for Children

Áreas de especialidad

  • Hematología pediátrica y adultos
  • Diagnóstico de laboratorio y hemostasia
  • Enfermería
  • Cirugía y cuidados ortopédicos
  • Fisioterapia
  • Psicología/Servicios sociales

Estados Unidos – Nueva York, Nueva York

Año de designación del CIEHEMO: 1993

Mount Sinai School of Medicine
Mount Sinai Medical Center

Áreas de especialidad

  • Hematología pediátrica y adultos
  • Diagnóstico de laboratorio y hemostasia
  • Enfermería
  • Cirugía y cuidados ortopédicos
  • Genética molecular
  • Servicios sociales

Estados Unidos - Rochester, Minnesota

Año de designación del CIEHEMO: 1974

Mayo (Rochester) Comprehensive Hemophilia Center, and International Hemophilia Training Center (WFH)
Mayo Clinic/Foundation and Mayo Medical School

Áreas de especialidad

  • Hematología pediátrica y adultos
  • Banco de sangre
  • Diagnóstico de laboratorio y hemostasia
  • Genética molecular
  • Enfermería
  • Cirugía y cuidados ortopédicos
  • Fisioterapia
  • Psicología/Servicios sociales

Estados Unidos - San Diego, California

Año de designación del CIEHEMO: 2018

Hemophilia & Thrombosis Treatment Center
University of California San Diego

Áreas de especialidad

  • Hematología adultos
  • Fisioterapia/Terapia ocupacional
  • Cuidados dentales
  • Cuidados ortopédicos (sin cirugía)
  • Diagnóstico de laboratorio
  • Enfermería
  • Psicología/Servicios sociales

Reino Unido – Londres

Año de designación del CIEHEMO: 1970

Katharine Dormandy Haemophilia Centre and Thrombosis Unit
Royal Free Hospital

Áreas de especialidad

  • Hematología adultos
  • Diagnóstico de laboratorio y hemostasia
  • Enfermería
  • Cirugía y cuidados ortopédicos
  • Fisioterapia/Terapia ocupacional
  • Genética molecular
  • Psicología/Servicios sociales

Reino Unido – Oxford

Año de designación del CIEHEMO: 1972

Oxford Haemophilia and Thrombosis Centre
Churchill Hospital

Áreas de especialidad

  • Hematología pediátrica y adultos
  • Diagnóstico de laboratorio y hemostasia
  • Cirugía y cuidados ortopédicos
  • Enfermería
  • Genética molecular
  • Fisioterapia/Terapia ocupacional
  • Psicología y servicios sociales

Reino Unido - Sheffield

Año de designación del CIEHEMO: 1992

Sheffield Haemophilia and Thrombosis Centre
Royal Hallamshire Hospital

Áreas de especialidad

  • Hematología adultos
  • Diagnóstico de laboratorio y hemostasia
  • Genética molecular

Tailandia - Bangkok

Año de designación del CIEHEMO: 1984

International Hemophilia Training Center – Bangkok
Ramathibodi Hospital
Mahidol University

Áreas de especialidad

  • Hematología pediátrica y adultos
  • Banco de sangre
  • Cuidados y cirugía dentales
  • Diagnóstico de laboratorio y hemostasia
  • Enfermería
  • Cirugía y cuidados ortopédicos
  • Fisioterapia
  • Psicología y servicios sociales

Suecia - Malmö

Año de designación del CIEHEMO: 1976

Malmö Centre for Thrombosis and Haemostasis
Department for Hematology and Coagulation Disorders, Skåne University Hospital
Skåne University Hospital

Áreas de especialidad

  • Hematología pediátrica y adultos
  • Banco de sangre (a pedido)
  • Cuidados y cirugía dentales
  • Diagnóstico de laboratorio y genética
  • Enfermería
  • Cirugía y cuidados ortopédicos
  • Fisioterapia

España - Madrid

Año de designación del CIEHEMO: 2018

Hospital Universitario La Paz

Áreas de especialidad

  • Hematología pediátrica y adultos
  • Fisioterapia/Terapia ocupacional
  • Cuidados dentales
  • Cuidados ortopédicos (sin cirugía)
  • Diagnóstico de laboratorio
  • Enfermería
  • Psicología/Servicios sociales

España - Valencia

Año de designación del CIEHEMO: 1997

Haemostasis and Thrombosis Unit
Haematology Service, University and Polytechnic Hospital

Áreas de especialidad

  • Hematología pediátrica y adultos
  • Diagnóstico de laboratorio y hemostasia
  • Genética molecular
  • Enfermería
  • Cirugía y cuidados ortopédicos
  • Fisioterapia
  • Banco de sangre
  • Psicología y servicios sociales

Sudáfrica - Johannesburgo

Año de designación del CIEHEMO: 2016

Haemophilia Comprehensive Care Centre
Charlotte Maxeke Johannesburg Academic Hospital

Áreas de especialidad

  • Hematología pediátrica y adultos
  • Cuidados dentales
  • Diagnóstico de laboratorio y hemostasia
  • Enfermería
  • Cirugía ortopédica
  • Fisioterapia

Singapur - Singapur

Año de designación del CIEHEMO: 2002

Singapore General Hospital Haemophilia Treatment Clinic

Áreas de especialidad

  • Hematología adultos
  • Diagnóstico de laboratorio
  • Enfermería
  • Cuidados dentales
  • Cuidados ortopédicos (sin cirugía)
  • Fisioterapia
  • Psicología

Senegal - Dakar

Año de designación del CIEHEMO: 2016

Centre International de Traitement de l’Hémophilie (CITH) de Dakar
Centre National de Transfusion Sanguin (CNTS)

Áreas de especialidad

  • Hematología pediátrica y adultos
  • Diagnóstico de laboratorio y hemostasia
  • Enfermería
  • Banco de sangre
  • Fisioterapia

Rusia – Moscú y San Petersburgo

Año de designación del CIEHEMO: 2010

National Medical Research Center of Hematology of the Russian Academy of Medical Sciences

Áreas de especialidad

  • Hematología adultos
  • Enfermería
  • Cirugía ortopédica

Países Bajos - Utrecht

Año de designación del CIEHEMO: 1996

Van Creveldkliniek
University Medical Centre Utrecht

Áreas de especialidad

  • Hematología pediátrica y adultos
  • Diagnóstico de laboratorio y hematología
  • Enfermería
  • Cirugía y cuidados ortopédicos
  • Fisioterapia/Terapia ocupacional
  • Psicología y servicios sociales

Japón - Nara

Año de designación del CIEHEMO: 1998

Nara Hemophilia Centre
Nara Medical University

Áreas de especialidad

  • Hematología pediátrica y adultos
  • Cuidados dentales
  • Diagnóstico de laboratorio y genética molecular
  • Enfermería
  • Cirugía y cuidados ortopédicos
  • Fisioterapia

Italia - Milán

Año de designación del CIEHEMO: 1972

Angelo Bianchi Bonomi Hemophilia and Thrombosis Centre
Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico

Áreas de especialidad

  • Hematología pediátrica y adultos
  • Banco de sangre
  • Cuidados dentales
  • Valoración diagnóstica de defectos de la función plaquetaria
  • Diagnóstico de laboratorio y hemostasia
  • Genética molecular
  • Enfermería
  • Cirugía y cuidados ortopédicos
  • Fisioterapia
  • Psicología y servicios sociales

Israel - Tel Hashomer

Año de designación del CIEHEMO: 1993

National Hemophilia Center, Tel Hashomer
Sheba Medical Center

Áreas de especialidad

  • Hematología pediátrica y adultos
  • Cirugía ortopédica
  • Enfermería
  • Fisioterapia
  • Cuidados dentales
  • Diagnóstico de laboratorio (sin genética)
  • Psicología

India - Mumbai

Año de designación del CIEHEMO: 2000

National Institute of Immunohaematology (ICMR) and KEM Hospital

Áreas de especialidad

  • Hematología pediátrica y adultos
  • Diagnóstico de laboratorio y hemostasia
  • Genética molecular
  • Cuidados dentales
  • Enfermería
  • Cuidados ortopédicos
  • Fisioterapia

India - Vellore

Año de designación del CIEHEMO: 2000

Christian Medical College, Vellore

Áreas de especialidad

  • Hematología pediátrica y adultos
  • Diagnóstico de laboratorio y hemostasia
  • Genética molecular con diagnóstico prenatal
  • Enfermería
  • Manejo musculoesquelético – Terapia física y ocupacional con fisiatría
  • Cirugía y cuidados ortopédicos
  • Banco de sangre
  • Cuidados dentales
  • Psicología/Servicios sociales
  • Diagnóstico prenatal

París - Francia

Año de designación del CIEHEMO: 1972

Paris Haemophilia Centre (Necker (N) and Kremlin Bicêtre (KB) Hospitals)

Áreas de especialidad

  • Hematología pediátrica y adultos
  • Diagnóstico de laboratorio y hemostasia
  • Enfermería
  • Cuidados dentales
  • Cirugía y cuidados ortopédicos
  • Fisioterapia
  • Psicología y servicios sociales

Egipto – El Cairo

Año de designación del CIEHEMO: 2015

Shabrawishi Hospital, Egyptian Society of Hemophilia, and Cairo University

Áreas de especialidad

  • Hematología pediátrica y adultos
  • Diagnóstico de laboratorio y hemostasia
  • Enfermería
  • Genética molecular
  • Banco de sangre
  • Fisioterapia

Canadá - Toronto

Año de designación del CIEHEMO: 2018

University of Toronto Hemophilia Centres
St. Michael’s Hospital

Áreas de especialidad

  • Servicios hematológicos para adultos
  • Servicios de hematología pediátrica
  • Fisioterapia/Terapia ocupacional
  • Cuidados dentales
  • Cuidados ortopédicos (sin cirugía)
  • Diagnóstico de laboratorio
  • Enfermería
  • Psicología/Servicios sociales

Brasil - Campinas, SP

Año de designación del CIEHEMO: 2008

Hemocentro – “Unidade de Hemofilia Cláudio Luiz Pizzigatti Corrêa”
Hemocentro Unicamp, University of Campinas

Áreas de especialidad

  • Hematología pediátrica y adultos
  • Diagnóstico de laboratorio y genética molecular
  • Enfermería
  • Odontología
  • Cirugía y cuidados ortopédicos
  • Fisioterapia

Bélgica - Bruselas

Año de designación del CIEHEMO: 2013

Haemostasis and Thrombosis Unit
Division of Haematology
Cliniques universitaires Saint-Luc

Áreas de especialidad

  • Hematología pediátrica y adultos
  • Diagnóstico de laboratorio
  • Cirugía y cuidados ortopédicos
  • Fisioterapia

Austria - Viena

Año de designación del CIEHEMO: 1972

Medical University of Vienna
Department of Medicine I
Clinical Division of Hematology and Hemostaseology

Áreas de especialidad

  • Hematología pediátrica y adultos

Australia - Melbourne

Año de designación del CIEHEMO: 2005

Ronald Sawers Haemophilia Treatment Centre

Áreas de especialidad

  • Hematología adultos
  • Diagnóstico de laboratorio y hemostasia
  • Cirugía y cuidados ortopédicos
  • Enfermería
  • Cuidados dentales
  • Banco de sangre
  • Psicología
  • Trabajo social

Argentina - Buenos Aires

Año de designación del CIEHEMO: 1974

Instituto Asistencial “Dr. Alfredo Pavlovsky”
(Fundación de la Hemofilia)

Instituto De Investigaciones Hematológicas “Dr. Mariano R. Castex” (IIHEMA
(Academia Nacional de Medicina)

Áreas de especialidad

  • Hematología pediátrica y adultos
  • Fisioterapia
  • Cuidados dntales
  • Cirugía y cuidados ortopédicos
  • Diagnóstico de laboratorio y diagnóstico genético
  • Enfermería
  • Psicología
  • Servicios sociales

Nuestra historia

Frank Schnabel, un hombre de negocios canadiense con hemofilia A grave, fundó la FMH en 1963. Schnabel estaba convencido de que las organizaciones de pacientes podían ser mucho más eficaces, y hacer más para representar los intereses de las personas con trastornos de la coagulación, si trabajaban unidas. El primer Congreso de la FMH se realizó en Copenhague, Dinamarca, el 25 de junio de 1963, y en él participaron representantes de 12 países. Actualmente, la FMH y su red mundial de organizaciones nacionales miembros (ONM) representan los intereses de personas con hemofilia y otros trastornos de la coagulación hereditarios en 151 naciones.