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UnknownX-linked myotubular myopathy: A prospective international natural history study.
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S-EPMC6550499 | biostudies-literature
UnknownHierarchical Bayesian modelling of disease progression to inform clinical trial design in centronuclear myopathy.
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S-EPMC7789189 | biostudies-literature
UnknownThe 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study.
Not available
S-EPMC3824082 | biostudies-literature
UnknownThe 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study.
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S-EPMC3826053 | biostudies-literature
UnknownSeven-Year Experience From the National Institute of Neurological Disorders and Stroke-Supported Network for Excellence in Neuroscience Clinical Trials.
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S-EPMC7483960 | biostudies-literature
UnknownDefining the clinical, molecular and imaging spectrum of adaptor protein complex 4-associated hereditary spastic paraplegia.
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S-EPMC7780481 | biostudies-literature
UnknownAtaluren treatment of patients with nonsense mutation dystrophinopathy.
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S-EPMC4241581 | biostudies-literature
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