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FDA Scientists Raise Questions About Duchenne Drug
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Fusion42 · 27 July 2026 · Fusion42 review
FDA scientists challenge clinical trial data for deramiocel, a stem cell therapy for Duchenne muscular dystrophy, stating the drug failed to meet its primary endpoint in phase III trials despite researchers' contrary claims presented at the American Academy of Neurology annual meeting.
This Wire brief sits within Fusion42's coverage of Biotech.
◆ ◆ The Wire takeaway
The FDA is now calling out trial data inconsistencies in public before approval decisions, not after. Your cell therapy's endpoint success is worthless if your statistical methods don't hold—regulators will interrogate your trial design in the light, and they will find it.
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1 source · 27 Jul 2026
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