Medical and Scientific News
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Critical need for harmonized post-marketing surveillance in gene therapy
With regulatory approval of the first gene therapy for clinical use outside of clinical trials anticipated in mid-2020, there is an urgent need to collect...
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Findings from a CHS “Dream of a Cure” research project now published
Findings from a two-year research project on inherited platelet function disorders led by Dr. Catherine Hayward of McMaster University as principal investigator, funded by a...
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Dangerously high FIX levels reported in hemophilia B gene therapy
Montreal – June 29, 2020 – During the question-and-answer period of a session on gene therapy during the World Federation of Hemophilia’s virtual summit (June...
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Biomarin’s gene therapy for hemophilia A to be called Roctavian
Montreal, May 14, 2020 – Biomarin announced on May 11 its gene therapy currently awaiting approval by the U.S. Food and Drug Administration will be...
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Gene Therapy Updates
FDA grants priority review for Biomarin’s hemophilia A gene therapy San Rafael, California, February 21, 2020 – Biomarin Pharmaceuticals has announced that the U.S. Food...