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Hemophilia Bleeding Disorder Industry News
Industry News

UniQure Announces New Data in Phase I/II Clinical Trial

______________________________________________________________________________ Note: The following is edited from a press release from UniQure. Read the full press release in it’s entirety here. UniQure announced new and updated results during the American Society of Hematology (ASH) meeting in relation to its ongoing, dose-ranging Phase I/II trial of AMT-060. AMT-060 is UniQure’s proprietary, investigational gene therapy for patients with severe […]

Industry News

Sangamo Biosciences Announces Plans to File for New Investigational Drug

______________________________________________________________________________ Note: The following is edited from a press release from Sangamo Biosciences. Read the full press release in its entirety here. Sangamo Biosciences presented data during the recent American Society of Hematology (ASH) Annual Meeting regarding preclinical and manufacturing data of SB-525, its gene therapy program for hemophilia A. “We have developed an improved […]

Industry News

Spark Therapeutics Shares Updated Phase I/II Clinical Trial Data

______________________________________________________________________________ Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in it’s entirety here.. Spark Therapeutics and Pfizer announced updated results from the Phase 1/2 study of SPK-9001, an investigational recombinant gene transfer product in hemophilia B, at the 58th American Society of Hematology (ASH) Annual Meeting, […]

Industry News

Alnylam Presents Phase I Clinical Drug Trial Data at ASH 2016

  Note: The following is an excerpt from a press release from Alnylam Pharmaceuticals, Inc. regarding news from their recent presentation at the American Society of Hematology (ASH) Annual Meeting. The original article can be read in its entirety here. ______________________________________________________________________________ Alnylam Pharmaceuticals, Inc. presented data from the phase 1 study of fitusiran at the […]

Industry News

Octapharma Announces Two Hemophilia A Clinical Trials Utilizing Wilate

The following is excerpted from a press release from Octapharma USA. Read the press release in it’s entirety here.  Octapharma USA has announced that WILATE®, von Willebrand Factor/Coagulation Factor VIII Complex (Human) Lyophilized Powder for Solution for Intravenous Injection will be utilized for two important clinical trials for hemophilia A launching in December. Octapharma USA […]

Industry News

Prescription Benefit Program Changes for Recombinant Clotting Factor Product

  It has come to our attention that Helixate FS, a recombinant clotting factor product sold and distributed by CSL Behring, is being removed from prescription benefit programs for a limited number of patients using CVS/Caremark effective January 1, 2017. Patients currently using Helixate FS who have the “Advanced Control Formulary” plan with CVS/Caremark will have the […]

Industry News

PPTA Releases Updated Statement on Zika

The following is a summary of a statement released by the Plasma Protein Therapeutics Association (PPTA). To read the PPTA statement in full, click HERE. On September 8, 2016, PPTA issued an updated news release regarding the Zika virus (ZIKV) and plasma protein therapies. In the release, PPTA notes that: ZIKV is in a family of (lipid-enveloped) viruses that is […]

Industry News

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

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 […]

Industry News

Baxalta Sponsors 63 Students in 2016-17

Baxalta, now part of Shire, has awarded scholarships to 63 students from 13 states, further adding to the list of recipients of its Education Advantage scholarship program. For the seventh consecutive year, Baxalta US Inc. is helping eligible students with a bleeding disorder offset the costs of higher education, advance their careers, and pursue their […]

Industry News

First FDA-Approved Recombinant von Willebrand Factor, Vonvendi, Now Available for von Willebrand Disease

The following is excerpted from a press release from Baxalta, now a part of Shire. Read the press release in it’s entirety here. Shire has announced that Vonvendi [von Willebrand factor (Recombinant)], indicated for the on-demand treatment and control of bleeding episodes in adults (age 18 and older) diagnosed with von Willebrand disease, is now […]

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