Cette section du site Internet de la Fédération mondiale de l’hémophilie (FMH) est réservée aux laboratoires pharmaceutiques fabricant des médicaments et autres produits traitant les troubles de la coagulation afin de diffuser les communiqués de presse susceptibles d’intéresser les membres de notre communauté.
La FMH ne cautionne en aucun cas les informations diffusées dans la présente section et ne soutient aucune alternative thérapeutique en particulier. Les déclarations et les opinions exprimées ici ne reflètent en aucun cas ni le point de vue, ni la politique de la FMH et ne constituent en aucun cas des recommandations de sa part.
Les communiqués de presse des entreprises partenaires sont disponibles uniquement en anglais.
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
Le Programme d’aide humanitaire de la FMH permet de combler l’écart dans l’accès aux soins et aux traitements en fournissant le soutien indispensable dont ont besoin les ONM, les centres de traitement de l’hémophilie et les professionnels de santé dans les pays émergents.
Restez informé des toutes dernières actualités de la communauté des troubles de la coagulation, des avancées médicales aux événements incontournables.
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.
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.
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.
National Hemophilia Center, Tel Hashomer
Sheba Medical Center
Haemophilia Comprehensive Care Centre
Charlotte Maxeke Johannesburg Academic Hospital
Singapore General Hospital Haemophilia Treatment Clinic
Centre International de Traitement de l’Hémophilie (CITH) de Dakar
Centre National de Transfusion Sanguin (CNTS)
National Medical Research Center of Hematology of the Russian Academy of Medical Sciences
University Medical Centre Utrecht
Nara Hemophilia Centre
Nara Medical University
Angelo Bianchi Bonomi Hemophilia and Thrombosis Centre
Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico
National Institute of Immunohaematology (ICMR) and KEM Hospital
Christian Medical College, Vellore
Malmö Centre for Thrombosis and Haemostasis
Department for Hematology and Coagulation Disorders, Skåne University Hospital
Skåne University Hospital
Paris Haemophilia Centre (Necker (N) and Kremlin Bicetre (KB) Hospitals)
Shabrawishi Hospital, Egyptian Society of Hemophilia, and Cairo University
University of Toronto Hemophilia Centres
St. Michael’s Hospital
Hemocentro – “Unidade de Hemofilia Cláudio Luiz Pizzigatti Corrêa”
Hemocentro Unicamp, University of Campinas
Haemostasis and Thrombosis Unit
Division of Haematology
Cliniques universitaires Saint-Luc
Hemofiliecentrum Leuven, University of Leuven
Medical University of Vienna
Department of Medicine I
Clinical Division of Hematology and Hemostaseology
Ronald Sawers Haemophilia Centre
Instituto Asistencial “Dr. Alfredo Pavlovsky”
(Fundación de la Hemofilia)
Instituto De Investigaciones Hematológicas “Dr. Mariano R. Castex” (IIHEMA
(Academia Nacional de Medicina)
Hospital Universitario La Paz
Haemostasis and Thrombosis Unit
Haematology Service, University and Polytechnic Hospital
International Hemophilia Training Center – Bangkok
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.
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.
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.
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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.
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.
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.”
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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.
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.
|2006||Dr Bruce Evatt
|2012||Piet de Kleijn
|2010||Dr Mammen Chandy
|2008||Dr Man-Chiu Poon
|2006||Dr Norma de Bosch
|2004||Dr Parttraporn Insarangkura
Dr Carol K. Kasper
Katharine Dormandy Haemophilia Centre and Thrombosis Unit
Royal Free Hospital
Manchester Haemophilia Comprehensive Care Centre (Children’s and Adult’s)
Manchester Royal Infirmary and Manchester Children’s Hospital
Oxford Haemophilia Centre and Thrombosis Centre
Sheffield Haemophilia and Thrombosis Centre
Royal Hallamshire Hospital
|2020||Aluva (Inde) – Newcastle (Royaume-Uni))|
|2019||Yogyakarta / Solo (Indonésie) – Utrecht (Pays-Bas)|
|2018||Abidjan (Côte d’Ivoire) – Bruxelles (Belgique)|
|2017||Santa Cruz de la Sierra (Bolivie) – Buenos Aires (Argentine)|
|2016||Kampala (Ouganda) – Londres (Royaume-Uni)|
|2015||Varna (Bulgarie) – Bonn (Allemagne)|
|2014||Arequipa (Pérou) – Los Angeles (États‑Unis)|
|2013||Davangere (Inde) – Liverpool (Royaume-Uni)
Ludhiana (Inde) – Detroit (États‑Unis)
|2012||Hanoï (Vietnam) – Melbourne (Australie)|
|2011||Manado (Indonésie) – Utrecht (Pays-Bas)|
|2010||Delhi (Inde) – Winnipeg (Canada)
Yaoundé (Cameroun) – Genève (Suisse)
|2009||Chisinau (Moldavie) – Varsovie (Pologne)
Colombo (Sri Lanka) – Vellore (Inde)
|2008||Gaborone (Botswana) – Philadelphie (États-Unis)|
|2007||Vientiane (Laos) – Brest (France)
Damas (Syrie) – Montpellier (France)
|2006||Lima (Pérou) – Fort Worth (États-Unis)|
|2005||Erevan (Arménie) – Minneapolis (États-Unis)
Casablanca (Maroc) – Caen (France)
|2004||Beyrouth (Liban) – Genève (Suisse)
Saint-Domingue (République dominicaine) – Caracas (Venezuela)
|2003||Le Caire (Égypte) – Knoxville (États-Unis)|
|2002||Moscou (Russie) – Liverpool (Royaume-Uni)
Panama - Valence (Espagne)
|2001||Bangalore (Inde) – Saint-Louis (États-Unis)|
|2000||Pune (Inde) – Bradford (Royaume-Uni)|
|1999||Montevideo (Uruguay) – Buenos Aires (Argentine)
Tianjin (Chine) – Calgary (Canada)
|1998||Plovdiv (Bulgarie) – Bonn (Allemagne)|
|1997||Bogota (Colombie) – Los Angeles (États-Unis)
Tartu (Estonie) – Stockholm (Suède)
|1996||Timisoara (Roumanie) – Munich (Allemagne)
Riga (Lettonie) – Munster (Allemagne)
|1995||Klaipeda (Lituanie) – Malmö (Suède)|
|1994||Bratislava (Slovaquie) – Tel Hashomer (Israël)|
|2019||Dr Adolfo Llinás
|2012||Piet de Kleijn
|2011||Dr Lily Heijnen
|2010||Dr Horacio Caviglia
|2009||Jerome D. Wiedel
|2008||Dr Federico Fernández-Palazzi
|2005||Dr Marvin Gilbert
Yangzhou College, Xuzhou Medical University
|The effects of intra-articular injection of TNF-alpha Antagonists in treatment of haemophilic synovitis|
|Association entre la cinétique multisegment du pied et les lésions articulaires induites par le sang chez les patients adultes atteints d’arthropathie hémophile de la cheville|
|Decrease in joint inflammation, swelling and pain after a swimming protocol in animal model of experimental-induced hemarthrosis|
|2015||SM Javd Mortazavi
|Bilateral total knee arthroplasty in patients with hemophilia: A safe and cost-effective procedure|
|2013||Lize F.D. van Vulpen
|Une protéine de fusion d’IL4 et d’IL10 est également efficace pour protéger le cartilage contre les dommages induits par le sang par rapport aux composants individuels|
|Chirurgie pseudotumorale chez les patients atteints d’hémophilie A: résultats comparatifs entre patients inhibiteurs et non inhibiteurs|
Monique van Meegeren
|L’administration précoce d’IL-4 et d’IL-10 empêche les lésions cartilagineuses induites par le sang in vitro|
|Influence d’un protocole d’entraînement aquatique dans la réduction du risque de morbidité et de mortalité cardiovasculaires chez un hémophile|
|Impact de l’arthropathie de la cheville avec l’hémophilie sur l’incapacité de marche: analyse des variables énergétiques et mécaniques|
|2008||Jose Alberto Tlacuilo-Parra
|Marqueurs de la rotation osseuse et de la densité minérale osseuse chez les enfants atteints d’hémophilie|
|Les capacités de protection de l’interleukine-10 dans les dommages du cartilage induits par le sang|
|2006||Axel Seuser et E. Kusch
|Etude multicentrique du résultat orthopédique des membres inférieurs chez 249 enfants atteints d’hémophilie|
|2005||Frank van Genderen, Nico van Meeteren, Lily Heijnen, Piet de Kleijn, Marijke van den Berg, Paul Helders
|Liste de contrôle des activités d’hémophilie: développement final et validation d’un questionnaire d’auto-évaluation spécifique à l’hémophilie sur les capacités fonctionnelles|
|2004||Goris Roosendaal, Zalima N Jahangier, Kim MG Jacobs, Johannes WJ Bijlsma, Floris PJG Lafeber
|Radiosynoviorthesis avec YTTRIUM-90 résultats dans les effets directs défavorables sur le cartilage: une étude humaine in vitro|
|2003||Axel Seuser, Thomas Wallny, Günter Schumpe, Elmar Trunz, Hans-Hermann Brackman
|Comment conseillez-vous aux jeunes hémophiles de trouver le bon sport? Un nouvel algorithme sûr|
|2002||James Luck, Jr. et Mauricio Silva
|Examen à long terme d’arthroplastie totale du genou|
|Chlorhydrate d’Oxytetracycline: un nouveau matériel pour les orthèses synoviales chimiques|
|1999||William J. Ribbans et J.L. Hicks
|Communication et coopération pour le nouveau millénaire: l’avenir du comité musculo-squelettique au XXIe siècle|
|1998||Adolfo Llinas, Mauricio Silva, Monica Duarte, Gonzalo Ucros, Graciela Perini, Angela Maria Cerquera, Andres Forero
|La synoviorthèse au phosphate chromique sans âge devrait être la première option de traitement pour la synovite hémophile chronique active|
|2012||Martha Patricia Monteros Rincon
|2010||Antonia Luque de Garrido
|2008||Jean Michel Bouchez|