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FDA allows distribution of muscular dystrophy drug again after public criticism
The agency’s now-reversed decision to halt distribution of a gene therapy for children suffering from muscular dystrophy ...
FDA Suspends Muscular Dystrophy Drug After 3 Patient Deaths
The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...
US FDA's chief medical and science officer Prasad departs agency
Vinay Prasad, the U.S. Food and Drug Administration's chief medical and science officer, has left the health regulator, the U ...
Fierce Pharma1d
FDA takes U-turn on Sarepta's Elevidys, backing Duchenne gene therapy again in ambulatory patients
Monday, the FDA said Sarepta may resume treating ambulatory DMD patients with Elevidys. The recommendation comes just 10 days ...
FDA Probes Death Of 8-Year-Old Who Received Sarepta's Elevidys
FDA probes Elevidys after an 8-year-old's death in Brazil; Sarepta and Roche say the death was unrelated to gene therapy ...
Opinion
On Sarepta, a Welcome FDA Reversal
After our editorial, the agency relents to allow a Duchenne treatment.
FDA opens probe into mysterious death linked to gene therapy
The Food & Drug Administration is investigating the death of an 8‑year‑old following Elevidys gene therapy for Duchenne ...
CBS News8y
First drug for Duchenne muscular dystrophy, Exondys 51, gets FDA ...
The FDA cleared Sarepta Therapeutics’ Exondys 51 for a rare form of Duchenne muscular dystrophy, a deadly inherited disease that affects boys.