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 Sigilon Therapeutics. Read the press release on the Sigilon website聽here. Sigilon Therapeutics聽has announced it has received Orphan Drug Designation for SIG-001, an investigational therapy for hemophilia A that leverages Sigilon鈥檚 Shielded Living Therapeutics™聽platform to implant cells engineered to produce stable blood plasma levels of factor VIII, a crucial […]
Note: The following is an edited version of a press release originally published by the US Food and Drug Administration (FDA). The original release can be read here. The U.S. Food and Drug Administration announced on Tuesday, October 20, 2015, the approval of 聽Coagadex, Coagulation Factor X (Human), for hereditary Factor X (10) deficiency. Until […]
This is an edited version of a press release from CSL Behring. To read the full release, please聽click here. ____________________ 聽CSL Behring聽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 […]
Alnylam Pharmaceuticals, Inc. (ALNY), a leading RNAi therapeutics company, announced today that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has granted Orphan Drug Designations for ALN-AT3 as an orphan medicinal product for the treatment of hemophilia A and hemophilia B. Alnylam is developing ALN-AT3, a subcutaneously administered RNAi therapeutic targeting antithrombin […]
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