Very low residual dystrophin quantity is associated with milder dystrophinopathy

de Feraudy Y, Yaou RB, Wahbi K, et al. Very low residual dystrophin quantity is associated with milder dystrophinopathy. Annals of neurology 2020   Abstract OBJECTIVE: To determine whether a low residual quantity of dystrophin protein is associated with delayed clinical milestones in patients with DMD mutations. METHODS: We performed a retrospective multicentric cohort study […]