priors.science/reviews/gene-therapy-neuromuscular-disease

Gene Therapy Neuromuscular Disease

The current evidence on 14 claims, ordered from most established to most contested. Each score is the panel’s evidence certainty — how firmly the literature supports the claim as stated.

14claims tracked
83primary papers reviewed
6 Jul 2026latest evidence review
Weeklyre-scored against new papers
2 Established · 7 Likely · 5 Uncertain · 0 Doubtful  |  four-reviewer panel · PICO Framework
SignalClaimStandingEvidence certaintyCorpus
StableOnasemnogene improves spinal muscular atrophy type 1 motor functionSma GTEstablished86%12
StableDelandistrogene produces micro-dystrophin expressionDMD GTEstablished85%5
StableHigh-dose adeno-associated virus carries serious hepatotoxicity riskRare NMDLikely80%6
StablePresymptomatic spinal muscular atrophy treatment superior to symptomaticSma GTLikely80%7
StableTransaminase elevation common manageable adverse eventAav SafetyLikely79%8
StablePre-existing adeno-associated virus antibodies limit gene therapy eligibilityAav SafetyLikely77%5
StableLimb-girdle muscular dystrophy gene therapy functional improvement limitedLgmd GTLikely76%5
StableAT132 gene therapy improves X-linked myotubular myopathy motor functionRare NMDLikely69%5
StableSarcoglycanopathy gene therapy shows protein restorationLgmd GTLikely68%6
StableOnasemnogene durable benefit beyond 5 yearsSma GTUncertain64%5
StableExon-skipping gene therapy restores partial dystrophinDMD GTUncertain62%6
StableGene therapy superior to nusinersen in spinal muscular atrophy type 1Sma GTUncertain62%4
StableIgG-cleaving enzymes enable seropositive patient accessAav SafetyUncertain59%5
StableDelandistrogene functional benefit in Duchenne muscular dystrophy uncertainDMD GTUncertain44%4
Standing — what the evidence certainty means
Established≥ 85%Strong, consistent evidence. Unlikely to change.
Likely65–84%Well supported, with some gaps or indirect evidence.
Uncertain40–64%Mixed or limited evidence. Genuinely open.
Doubtful15–39%Little support; the weight of evidence leans against it.
Refuted< 15%The evidence contradicts it — confidently false as stated.
Recent signal
strengthenedNew evidence raised the certainty since the last review.
weakenedNew evidence lowered the certainty since the last review.
newA claim added to the review recently.
StableNo recent change to the standing.
Standing, evidence certainty and corpus are always shown. The study behind each move, why it moved, and the sceptic’s challenge are delivered to subscribers.
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