3 ms·
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": > [Sa
by n4r9 2y ago
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-therapy-fails-to-meet-primary-endpoint-in-pivotal-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-elevidys-fda-approval-peter-marks-overruled-staff/ 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?