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. https://geneticliteracyproject.org/2020/01/08/will-2020-see-the-debut-of-promising-gene-therapy-for-hemophilia-a-its-up-to-the-fda/
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SHARESHARESHARE
WILL 2020 SEE THE DEBUT OF PROMISING GENE THERAPY FOR HEMOPHILIA A? IT’S UP TO THE FDA.
The clotting disorder hemophilia A may become the third gene therapy that the US Food and Drug Administration approves, joining treatments for a form of retinal blindness in 2017, and spinal muscular atrophy in 2019. Biomarin Pharmaceutical Inc. has submitted a biologics license application to FDA and documentation of clinical trial results to the European Medicines Agency, with reviews slated to begin early this year at both organizations. READ MORE