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by n4r9
725 days ago
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The links from OP are paywalled but the first few visible lines are enough to cast significant doubt for me on whether the treatment is even "promising": > [Sarepta's] gene therapy for Duchenne muscular dystrophy failed to improve muscle function compared to a placebo in a large clinical trial https://www.statnews.com/2023/10/30/sareptas-duchenne-gene-t... > the drug failed a large, Phase 3 trial last year ... three review teams ... wrote that the data Sarepta submitted "cast significant uncertainty regarding the benefits of the treatment". https://www.statnews.com/2024/06/20/sarepta-duchenne-elevidy... I'm not sure what makes this treatment "promising" other than the gushings of the CEO. Are you arguing that we should give new treatments a free pass on regulations as long as its for a death-sentence disease and the CEO has connections? |
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