4 ms·
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
by trehalose 2y ago
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.
- fastaguy88 2y agoAbsolutely. 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.