Late-stage study underway for BioMarin’s gene therapy for hemophilia A – Seeking Alpha
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Late-stage study underway for BioMarin’s gene therapy for hemophilia A
Seeking Alpha The first patient has been dosed in a Phase 3 clinical trial, GENEr8-1, assessing BioMarin Pharmaceutical’s (NASDAQ:BMRN) gene therapy valoctocogene roxaparvovec (VR) in patients with hemophilia A. The study is the first of two registration trials that … |
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HSS’ Dr. Christopher Mendias earns $800k grant for stem cell research — 5 notes
Becker’s Orthopedic & Spine The Orthopaedic Research and Education Foundation and National Stem Cell Foundation gave Christopher L. Mendias, PhD, of New York City-based Hospital for Special Surgery, a research grant. Here are five notes: 1. Dr. Mendias earned the OREF Clinical … |
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New Gene Therapies Could Change Hemophilia Forever
Motley Fool At last week’s American Society of Hemophilia conference, Spark Therapeutics (NASDAQ:ONCE) and BioMarin (NASDAQ:BMRN) presented promising data for gene therapy drugs that could change the way hemophilia is treated, making patients’ lives significantly … |
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FDA Approves Gene Therapy for Rare Form of Blindness
U.S. News & World Report TUESDAY, Dec. 19, 2017 (HealthDay News) — A new gene therapy to treat children and adults with a rare type of inherited vision loss has been approved by the U.S. Food and Drug Administration. It’s the first gene therapy approved in the United States … |