I hope it really works. Muscular dystrophy is such a bag of dicks. Poor kids.
this post was submitted on 23 Jun 2023
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So, Sarepta has multiple approved therapies that haven't reached positive clinical endpoints? How many times can the agency/drugmaker justify accelerated approval based on an unmet clinical need?
Imo, this just adds to the growing criticism of the agency's growing use of accelerated approvals without holding pharma companies to their post-approval obligations.
Interesting discussion in the article. Seems like it's a pretty experimental therapy atm without any conclusive evidence that it works.
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