Tag: FDA

A total of 126 posts are filed under FDA
BioPharma Dive: 5 Trends Shaping Rare Disease Drug Development
Story by Ned Pagliarulo Amid ongoing scrutiny of rising prescription drug costs, a three decade-old law has become a new flash point in the… Continue Reading
Designing Effective Rare Disease Registries
by Raymond A. Huml, MS, DVM, RAC and Margaret Dean, MBA, Quintiles MS at DDDmag.com   Rare disease research was once a neglected aspect of… Continue Reading
Teva Announces FDA Approval of AUSTEDO™ (deutetrabenazine) Tablets for the Treatment of Chorea Associated with Huntington’s Disease
Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA) today announced that the U.S. Food and Drug Administration (FDA) has approved… Continue Reading
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Call to Action: Endorsing Scott Gottlieb MD as Commissioner of Food and Drug Administration
Global Genes is joining the rare community to support Dr. Scott Gottlieb’s nomination to serve as Commissioner of Food and Drug… Continue Reading
CureDuchenne Applauds the FDA Approval of Marathon’s EMFLAZA (deflazacort) for the Treatment of Duchenne Muscular Dystrophy
EMFLAZA is the First Drug in the U.S. Approved for Use by Patients Age Five and Older with Duchenne Regardless of Genetic Mutation… Continue Reading
Trump’s Top Choices for FDA Could Bolster the ‘Right-to-Try’ Movement
By DYLAN SCOTT @dylanlscott   WASHINGTON — Advocates of “right-to-try” laws — giving terminally ill patients wider latitude to… Continue Reading
Lysogene Receives Rare Pediatric Disease Designation from FDA for LYS-GM101 for Treatment of GM1 Gangliosidosis
Lysogene, a leading, clinical-stage biotechnology company specializing in gene therapy for rare central nervous system diseases, today… Continue Reading
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