Hemophilia Federation of America is a national nonprofit organization that assists, educates and advocates for the bleeding disorders community.
The following is excerpted from a press release from PRNewswire. Read the press release in it’s entirety聽here. Sangamo BioSciences, Inc.聽announced earlier this week that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to SB-FIX, the company’s zinc finger nuclease (ZFN)-mediated genome editing product candidate for the treatment of hemophilia B. Sangamo […]
Note: The below is an edited version of a press release by UniQure.聽The original release can be read in it’s entirety聽here. On June 11, 2016,聽uniQure N.V. announced that additional data from its Phase I/II clinical trial of AMT-060 in hemophilia B patients were presented at the 21st聽Congress of the European Hematology Association (EHA) in Copenhagen, […]
Note: The below is an edited press release from Alnylam Pharmaceuticals, Inc. The original release can be read in its entirety聽here.聽 Alnylam Pharmaceuticals, Inc. announced on Monday, March 14, 2016, that it has initiated dosing of hemophilia patients with inhibitors in Part D of an ongoing Phase 1 clinical trial evaluating a once-monthly subcutaneous dose […]
Dear Addy, Several parents that I鈥檝e met at the HTC have recommended I should get my daughter a medical ID bracelet. Why is this so important? Are there resources available to help me get her one? Signed Inquiring About IDs Dear Inquiring, For patients with serious medical conditions like a bleeding disorder, it鈥檚 important that […]
Note: The below is an edited press release from Dimension Therapeutics, Inc. The original release can be read here. Dimension Therapeutics, Inc. announced on January 7 the initiation of a phase 1/2 study of DTX101, the company鈥檚 lead therapeutic candidate, for the treatment of patients with hemophilia B. DTX101 is designed to deliver stable expression […]
Dear Addy, I keep hearing about biosimilars. What are they, and how do they differ from generic drugs? Sincerely, Curious About Products Hi Curious, Thanks for your question. A biosimilar is a pharmaceutical drug that is made to have similar active properties as a biological drug that has already been licensed. Biologics are really important […]
Note: This is an edited form of a press release from Dimension Therapeutics, Inc. To read the original release in its entirety,聽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鈥檚 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.聽 New clinical data support the long-term safety and efficacy of ALPROLIX庐 [Coagulation Factor IX (Recombinant), Fc Fusion Protein] in people with severe hemophilia B treated for up to two years, Biogen announced on […]
Research showed that bleeding events were drastically decreased in animals with Hemophilia B. Using a viral vector to swap out faulty genes proved safe and could be used for the more common Hemophilia A. _______________________________ A multi-year, ongoing study suggests that a new kind of gene therapy for Hemophilia B could be safe and effective […]
This is an edited version of a press release from Biogen Idec. To read the full release, please聽click here. ____________________ Biogen Idec聽 and聽Swedish Orphan Biovitrum聽AB announced positive top-line results of the Kids B-LONG Phase 3 clinical study that evaluated the safety, efficacy and pharmacokinetics of ALPROLIX庐聽[Coagulation Factor IX (Recombinant), Fc Fusion Protein] in children under […]
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 […]
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