News

The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...
FDA probes Elevidys after an 8-year-old's death in Brazil; Sarepta and Roche say the death was unrelated to gene therapy ...
The U.S. Food and Drug Administration has approved PTC Therapeutics' oral drug to treat a rare inherited metabolic disorder ...
The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients ...
After initially refusing to suspend Elevidys distribution after two deaths, Sarepta has now given in to the FDA’s request, ...