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Call me cranky or old-fashioned, but I believe companies developing genetic medicines for even the rarest and most severe diseases should be conducting randomized, placebo-controlled clinical trials. Anything less is scientifically irresponsible and shows a lack of respect for patients and their families. These therapies are irreversible and carry substantial risks, so the benefits better be clearly demonstrated.
Yet at Neurogene, the developer of a gene therapy for Rett syndrome, expediency seems to be the priority. The company is collecting the least amount of data possible from single-arm, open-label studies that, by definition, will not demonstrate convincing proof of efficacy or safety.
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