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clock Created with Sketch. 15/01/21
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Originally posted by waynesworld
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A line in the sand appears to have been drawn. Gene therapy has been discredited as a foundational treatment in DMD. I don't think we can go back to the prevailing consensus that existed prior to the release of the Sarepta clinical trial data i.e. a transformative therapeutic benefit in DMD will require a significant increase in dystrophin production. Fortunately, for the boys and families in the DMD community we can go forward.
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@waynesworld , care to shed any light on where this trial fits into the Gene Therapy picture as it appeared in the latest MD news - seems they are excited about the news?Dosing Begins in Phase 3 Trial of Pfizer's Gene Therapy for Duchenne (musculardystrophynews.com)
