Tag: FDA

A total of 114 posts are filed under FDA
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RARECast: How to Improve Patient Access to Experimental Drugs
  Expanded access—a means by which physicians and patients can obtain experimental drugs outside of a clinical trial—has been an area… Continue Reading
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RARE DISEASE REPORT: FDA Approves HAE Drug Berinert For Pediatric Use
The FDA has approved Berinert [C1 Esterase Inhibitor (Human)], to be used in pediatric patients to treat Hereditary Angioedema (HAE)… Continue Reading
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Marathon Pharmaceuticals Announces Submission of Deflazacort New Drug Application to the FDA
Marathon Pharmaceuticals, LLC, a biopharmaceutical company developing treatments for rare diseases, today announced it has submitted a New… Continue Reading
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Intercept’s Liver Drug Wins FDA Approval To Treat Rare Disease
The FDA ended a busy week by giving a thumbs-up late Friday to obeticholic acid as a treatment for primary biliary cirrhosis, a rare… Continue Reading
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Rare Disease Report: FDA Expands Keytruda Indication for Advanced Melanoma
The FDA has approved an expanded indication for Keytruda (pembrolizumab), an anti-PD-1 (programmed death receptor-1) therapy, to include it… Continue Reading
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CureDuchenne Responds to FDA Advisory Committee Meeting on Drisapersen for Duchenne Muscular Dystrophy
CureDuchenne, the California-based nonprofit organization dedicated to finding cures for Duchenne muscular dystrophy, released the… Continue Reading
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FDA Awards KGI an Orphan Drug Designation for Rett Syndrome
A project that was part of Dr. Ian Phillips’ Writing an Orphan Drug Application (ALS 432) class recently led to the U.S. Food and Drug… Continue Reading