Autori: Comi GP

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Data

Ataluren treatment of patients with nonsense mutation dystrophinopathy.

Natural history of Type 1 spinal muscular atrophy: a retrospective, global, multicenter study

Clinical Phenotype of Pediatric and Adult Patients With Spinal Muscular Atrophy With Four SMN2 Copies: Are They Really All Stable?

Age, corticosteroid treatment and site of mutations affect motor functional changes in young boys with Duchenne Muscular Dystrophy

North Star Ambulatory Assessment changes in ambulant Duchenne boys amenable to skip exons 44, 45, 51, and 53: A 3 year follow up

The nonsense mutation stop+4 model correlates with motor changes in Duchenne muscular dystrophy

Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53

Timed Rise from Floor as a Predictor of Disease Progression in Duchenne Muscular Dystrophy: An Observational Study

6 Minute walk test in Duchenne MD patients with different mutations: 12 month changes

24 month longitudinal data in ambulant boys with Duchenne muscular dystrophy

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