Hemophilia Federation of America is a national nonprofit organization that assists, educates and advocates for the bleeding disorders community.
The following is a press release from Bayer regarding a voluntary recall. PATIENTS SHOULD NOT USE THIS PRODUCT! Bayer is voluntarily recalling two lots of Kogenate® FS antihemophilic factor (recombinant) 2000 IU vials in the United States to the patient level. Certain vials from these two lots that were labeled as Kogenate FS actually contain […]
Dear Bleeding Disorders Community — I want to let you know that I will be stepping down from HFA as your President and CEO at the end of August. Leaving HFA has been an incredibly difficult decision I have been wrestling with for about a year. I love HFA but ultimately this is just the […]
The following is an excerpt from a press release from Genentech. Read the press release in its entirety here. Genentech, a member of the Roche Group, announced today new data for Hemlibra® (emicizumab-kxwh) across multiple pivotal studies in people with hemophilia A with and without factor VIII inhibitors at the International Society on Thrombosis and […]
The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure, a gene therapy company advancing transformative therapies for patients with severe medical needs, announced updated clinical data on the three patients treated in the company’s ongoing Phase IIb study of AMT-061, an investigational AAV5-based gene […]
The following is an excerpt from a press release from Octapharma. Read the press release in its entirety here. Octapharma announced that new data on the benefits of Nuwiq® in patients with hemophilia A were presented during a scientific symposium at the 27th International Society on Thrombosis and Haemostasis Congress in Melbourne, Australia. The symposium, […]
The following is an excerpt from a press release from Sangamo. Read the press release in its entirety here. Sangamo Therapeutics Inc., a genomic medicine company, and Pfizer Inc. announced updated results from the Phase 1/2 Alta study evaluating investigational SB-525 gene therapy for severe hemophilia A. The data showed that SB-525 was generally well-tolerated […]
The following is an excerpt of a press release from Bayer. Read the press release in its entirety here. Bayer will present new data highlighting clinical outcomes from its hemophilia A portfolio, which include long-term data from the PROTECT VIII investigational study evaluating use of Jivi® antihemophilic factor (recombinant) PEGylated-aucl. These data will be presented […]
The following is an excerpt from a press release from BioMarin. Read the press release in its entirety here. BioMarin Pharmaceutical Inc. announced its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A achieved pre-specified clinical criteria for regulatory review in the U.S. and Europe. As of May 28, 2019, eight patients in […]
The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure announced updated clinical data in patients treated in the company’s ongoing Phase IIb study of AMT-061, an investigational AAV5-based gene therapy containing a patent-protected FIX-Padua variant, for the treatment of patients with severe and moderately severe hemophilia B. […]
The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure N.V., a gene therapy company, presented new preclinical data on its gene therapy candidates for the treatment of hemophilia A and for the treatment of Fabry disease during back-to-back oral presentations at the 22nd American Society […]
The following is from the FDA MedWatch. Read the recall notice in its entirety here. Alvogen Inc. is voluntarily recalling two lots of Fentanyl Transdermal System 12 mcg/h transdermal patches to the consumer level. A small number of cartons labeled 12 mcg/h Fentanyl Transdermal System patches contained 50 mcg/h patches. The 50 mcg/h patches that […]
The following is an excerpt from a press release from Sangamo Therapeutics. Read the press release in its entirety here. Sangamo Therapeutics Inc., a genomic medicine company, and Pfizer Inc. announced interim data from the Phase 1/2 Alta study evaluating investigational SB-525 gene therapy for severe hemophilia A. Data indicate that SB-525 was generally well-tolerated […]
The following is an excerpt from a press release from Spark Therapeutics. Read the press release in its entirety here. Spark Therapeutics , a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, announced today that it has entered into a definitive merger agreement for Roche to fully acquire Spark […]
The following is a press release from Roche. Read the press release in its entirety here. Roche announced it has joined the World Federation of Hemophilia Humanitarian Aid Program, a landmark initiative leading the effort to change the lack of access to care and treatment for people with inherited bleeding disorders in developing countries. Together […]
The following is an excerpt from a press release. Read the press release in its entirety here. Denmark-based Novo Nordisk received approval from the U.S. Food and Drug Administration for a new treatment for hemophilia A. However, the newly approved medication will not be available in the United States until 2020. Novo Nordisk said the […]
The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure, a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, announced updated clinical data in patients treated in the company’s ongoing Phase IIb study of AMT-061, an investigational AAV5-based gene therapy containing a […]
The following is an excerpt from a press release from Roche. Read it in its entirety here. Roche today announced that it has joined the World Federation of Hemophilia Humanitarian Aid Program, a landmark initiative leading the effort to change the lack of access to care and treatment for people with inherited bleeding disorders in […]
The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure, a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, today announced that it treated the first patient in its HOPE-B pivotal trial of AMT-061, an investigational AAV5-based gene therapy incorporating the […]
The following is a press release from Takeda, formerly Shire. Find the original press release here. Takeda Pharmaceutical Company Limited announced the completion of its acquisition of Shire, becoming a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan. Takeda now has an attractive, expanded geographic footprint and leading position in Japan and the U.S., bringing […]
The following is an excerpt from a press release from Save One Life Inc. Read the press release in its entirety here. Save One Life Inc. announces a new executive director. Save One Life is an international nonprofit that assists people with hemophilia in developing countries. Christopher G. Bombardier, famous for being the first person […]
The following is an excerpt from the Plasma Protein Therapeutics Association. Read the full press release here. The Board of Directors of the Plasma Protein Therapeutics Association announced Amy Efantis will be its next President and Chief Executive Officer effective Jan. 16, 2019. Having served most recently as Vice President, Global Public Policy & […]
The following is an excerpt from a press release from Genentech. Read the press release in its entirety here. Genentech, a member of the Roche Group announced data from the primary analysis of the Phase III HAVEN 2 study evaluating Hemlibra® prophylaxis in children younger than 12 years of age with hemophilia A with factor […]
The following is an excerpt from a press release from Sangamo. Read the press release in its entirety here. Hemophilia B is a rare, genetic bleeding disorder caused by a lack of the factor IX (FIX) protein, which is necessary for normal blood clotting. Current hemophilia B treatments typically require frequent intravenous infusions of clotting […]
The following is an excerpt from a press release from Genentech. Read the press release in its entirety here. Genentech, a member of the Roche Group, announced data from the primary analysis of the Phase III HAVEN 2 study evaluating Hemlibra® prophylaxis in children younger than 12 years of age with hemophilia A with factor VIII […]
The following is an excerpt from a press release from Bioverativ. Read the press release in its entirety here. Bioverativ Inc., a Sanofi company dedicated to transforming the lives of people with rare blood disorders, presented new data from the EXTEN-A Phase 1/2a trial of BIVV001 showing that a single 65 IU/kg dose of BIVV001 extended […]
The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure N.V., a gene therapy company advancing transformative therapies for patients with severe medical needs, announced updated results from its ongoing Phase I/II trial of AMT-060, and provided an update on AMT-061, the company’s next-generation gene therapy candidate […]
The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure announces the initial topline data from our Phase 2B dose-confirmation study of AMT-061, an investigational gene therapy for the treatment of patients with severe and moderately severe hemophilia B. All patients achieved and sustained therapeutic […]
The following is a press release from uniQure. uniQure, a gene therapy company advancing transformative therapies for patients with severe medical needs, announced the expansion of its research pipeline with novel AAV gene therapy approaches to treating Hemophilia A, Fabry disease and Spinocerebellar Ataxia Type 3 at the Company’s Research & Development Day held this […]
The following is an excerpt from a press release from uniQure. uniQure announced that they have treated the first patient in our Phase 2B dose-confirmation study of AMT-061, an investigational gene therapy for the treatment of patients with severe and moderately severe hemophilia B (see attached press release). “As a one-time administered therapy, AMT-061 has the […]
The following is an excerpt from a press release from Genentech. Read the press release in its entirety here. Genentech has announced new data that will be presented at this year’s annual American Society of Hematology meeting held in San Diego from Dec. 1-4. Ten Genentech medicines will be featured in more than 70 abstracts, including […]
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