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
> 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".
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?
> [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?