Eugenio Mercuri

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

Duchenne muscular dystrophy

Diagnosis and management of spinal muscular atrophy: Part 1: Recommendations for diagnosis, rehabilitation, orthopedic and nutritional care

Diagnosis and management of spinal muscular atrophy: Part 2: Pulmonary and acute care; medications, supplements and immunizations; other organ systems; and ethics

Mutations in the Fukutin-Related Protein Gene (FKRP) Cause a Form of Congenital Muscular Dystrophy with Secondary Laminin α2 Deficiency and Abnormal Glycosylation of α-Dystroglycan

Origin and timing of brain lesions in term infants with neonatal encephalopathy

Spinal muscular atrophy

Muscular dystrophies

Longitudinal effect of eteplirsen versus historical control on ambulation in <scp>D</scp> uchenne muscular dystrophy

Clinical and molecular genetic spectrum of autosomal dominant Emery-Dreifuss muscular dystrophy due to mutations of the lamin A/C gene

Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial