Tag: FDA

A total of 121 posts are filed under FDA
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
RDR: Generic Xyrem to Treat Narcolepsy Gets FDA Approval
The FDA has approved a generic version of Xyrem (sodium oxybate) Oral Solution, to treat cataplexy and excessive daytime sleepiness in… Continue Reading
RDR: FDA Approves Rubraca (rucaparib) for Rare Ovarian Cancer
The FDA has approved the orphan drug Rubraca (rucaparib) to treat women with advanced ovarian cancer who have been treated with 2 or more… Continue Reading
New Study Being Run for Catalyst Pharma for Patients with Congenital Myasthenic Syndromes (CMS)
Patients may now be eligible to participate in a new research study evaluating an investigational medicine for the treatment of CMS. WHY a… Continue Reading
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
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