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Sangamo BioSciences Receives Orphan Drug Designation From The FDA For SB-FIX

September 9, 2016

The following is excerpted from a press release from PRNewswire. Read the press release in it’s entirety here. Sangamo BioSciences, Inc. 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 […]

uniQure Announces Data from Phase I/II AMT-060 Clinical Trial

June 28, 2016

Note: The below is an edited version of a press release by UniQure. The original release can be read in it’s entirety here. On June 11, 2016, 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 Congress of the European Hematology Association (EHA) in Copenhagen, […]

Trial for SubQ Hemophilia Inhibitor Treatment Begins

March 14, 2016

Note: The below is an edited press release from Alnylam Pharmaceuticals, Inc. The original release can be read in its entirety here.  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: Why Are Medical Alert IDs Important?

January 15, 2016

Dear Addy, Several parents that I’ve 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’s important that […]

Dimension Therapeutics Announces Initiation of Phase 1/2 Study For Hemophilia B Treatment

January 7, 2016

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’s lead therapeutic candidate, for the treatment of patients with hemophilia B. DTX101 is designed to deliver stable expression […]

Dear Addy: Biosimilars

November 9, 2015

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 […]

FDA Approves Fast Track Designation for New Hemophilia B Treatment

September 18, 2015

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 […]

New Data Supports Safety & Efficacy Claims of ALPROLIX

August 17, 2015

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 […]

New Gene Therapy For Hemophilia Shows Potential As Safe Treatment

March 12, 2015

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 […]

Biogen Idec and Sobi See Progress in Pediatric Hemophilia B Study

February 27, 2015

This is an edited version of a press release from Biogen Idec. To read the full release, please click here. ____________________ Biogen Idec  and Swedish Orphan Biovitrum AB announced positive top-line results of the Kids B-LONG Phase 3 clinical study that evaluated the safety, efficacy and pharmacokinetics of ALPROLIX® [Coagulation Factor IX (Recombinant), Fc Fusion Protein] in children under […]

FDA Accepts CSL Behring’s Application for New Long-Acting Hemophilia B Product

February 4, 2015

This is an edited version of a press release from CSL Behring. To read the full release, please click here. ____________________  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 […]


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