November 28, 2020

The Niche

Knoepfler lab stem cell blog

Exondys 51

2 min read

The FDA caused major controversy by approving the drug Exondys 51 from Sarepta Therapeutics for treating Duchenne muscular dystrophy over the recommendations of a scientific panel it convened that had voted 7-3 against approval. Is this contentious Sarepta approval serve as a sign of how the FDA will deal with stem cells moving forward? Will the FDA approval, which was based on very limited, unclear data and in large part due to pressure from some patient advocates, serve as a warning to those of us hoping the agency …Read More