Re:Another drug approved despite review board
by trehalose on Monday June 24, 2024 @12:20PM
(#40777917)
Is production of a normal dystrophin transcript a real clinical endpoint, or just a biomarker that's supposed to correlate for one? It's not immediately obvious how normalizing production of a protein involved in muscle function is beneficial, if it doesn't actually improve muscle function.
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Re:Another drug approved despite review board
by fastaguy88 on Monday June 24, 2024 @04:56PM
(#40780669)
Absolutely. But DMD has a spectrum of severities, and it seems possible that for some patients, producing some normal transcript would make a real difference, so that until more people are treated, we cannot know when more normal transcripts make a difference.