Tag: FDA

A total of 116 posts are filed under FDA
FDA Helps Fund 21 Early Phase Trials for Rare Disease Treatments
(Source) by Zachary Brennan The US Food and Drug Administration (FDA) on Monday announced it is awarding $23 million in research grants for… Continue Reading
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Duchenne Community Rejoices over FDA Approval of Eteplirsen!
CureDuchenne applauds the first-ever approval of a drug in the U.S. to treat Duchenne muscular dystrophy. The drug, Exondys 51… Continue Reading
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
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
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
Keytruda 50mg/vial
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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