Journal: Neurology

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DMD Genotypes and Motor Function in Duchenne Muscular Dystrophy: A Multi-institution Meta-analysis With Implications for Clinical Trials

Safety and Efficacy of IV Onasemnogene Abeparvovec for Pediatric Patients With Spinal Muscular Atrophy: The Phase 3b SMART Study

Spinal Muscular Atrophy Functional Composite Score Revised (SMA-FCR) in Untreated and Nusinersen-Treated Patient Cohorts

Very early pattern of movement disorders in sepiapterin reductase deficiency

Neuromotor and cognitive outcomes of early treatment in tyrosine hydroxylase deficiency type B

Randomized, double-blind, placebo-controlled trial of phenylbutyrate in spinal muscular atrophy

Association Between Body Mass Index and Disability in Children With Charcot-Marie-Tooth Disease

The relationship between deficit in digit span and genotype in nonsense mutation Duchenne muscular dystrophy

Prevalence of Spinal Muscular Atrophy in the Era of Disease-Modifying Therapies: An Italian Nationwide Survey

Respiratory Trajectories in Type 2 and 3 Spinal Muscular Atrophy in the iSMAC Cohort Study

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