Hemophilia Federation of America is a national nonprofit organization that assists, educates, and advocates for the bleeding disorders community.
Note: the following is the edited form of a press releaseoriginally published by uniQure. The full release can be read here. On Thursday, January 7, 2016, uniQure N.V. announced preliminary topline results from the low-dose cohort of an ongoing Phase I/II clinical trial being conducted in adult hemophilia B patients treated with uniQure’s novel AAV5/FIX […]
Note: The following is an edited press release from Alnylam. The original release can be read in its entirety here. Alnylam Pharmaceuticals, Inc. announced on December 7, 2015 positive results from its ongoing Phase 1 clinical study with fitusiran (fi-TOO-si-ran), the recommended International Nonproprietary Name (INN) for ALN-AT3. Fitusiran is an investigational RNAi therapeutic targeting […]
Note: The following is the edited version of a press release issued by CSL Behring. The original release can be found here. On December 7, 2015, CSL Behring presented data from its Phase III PROLONG-9FP clinical program evaluating the efficacy and long-term safety of its investigational long-acting fusion protein linking recombinant coagulation factor IX with recombinant […]
Novo Nordisk, along with the Coalition for Hemophilia B, Hemophilia Federation of America, and National Hemophilia Foundation have announced the B-HERO-S (Bridging Hemophilia Experiences Results and Opportunities into Solutions) study open to all patients with hemophilia B and the caregivers of children with hemophilia B. About the Study Building on the success of the original […]
Note: This is an edited form of a press release from Dimension Therapeutics, Inc. To read the original release in its entirety, click here. Dimension Therapeutics, Inc. announced on Thursday, September 17 that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the company’s lead product candidate, DTX101, for the treatment of […]
Note: This is an edited form of a press release from Biogen. To read the original release in its entirety, click here. New clinical data support the long-term safety and efficacy of ALPROLIX® [Coagulation Factor IX (Recombinant), Fc Fusion Protein] in people with severe hemophilia B treated for up to two years, Biogen announced on […]
CSL Behring announced that the U.S. Food and Drug Administration (FDA) has accepted for review its Biologics License Application (BLA) for the marketing authorization of its long-acting fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP). Upon FDA approval, rIX-FP will provide hemophilia B patients with a long-acting treatment option with dosing intervals up […]
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