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Amedeo Smart

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Thornton CA, Moxley RT 3rd, Eichinger K, Heatwole C, et al. Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial. Lancet Neurol 2023;22:218-228.
PMID: 36804094


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