Ulrike Schara‐Schmidt

Guideline for the management of myasthenic syndromes

Heinz Wiendl, Angela Abicht, Andrew Chan et al. · Therapeutic Advances in Neurological Disorders · 2023 · 159 citations · Full text

Medicine, Immunology, Neurology +17

AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial

Jerry R. Mendell, Francesco Muntoni, Craig M. McDonald et al. · Nature Medicine · 2024 · 157 citations · Full text

Medicine, Molecular Genetics, Molecular Medicine +15

Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy

Michela Guglieri, Kate Bushby, Michael McDermott et al. · JAMA · 2022 · 123 citations · Full text

Pharmacy, Pharmacotherapy, Pediatric Clinical Pharmacology +16

Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

Eugenio Mercuri, Juan J. Vílchez, Odile Boespflug‐Tanguy et al. · The Lancet Neurology · 2024 · 120 citations

Medicine, Pediatrics, Pharmacotherapy +7

Effect of nusinersen on motor, respiratory and bulbar function in early-onset spinal muscular atrophy

Astrid Pechmann, Max Behrens, Katharina Dörnbrack et al. · Brain · 2022 · 70 citations · Full text

Medicine, Neurology, Pediatrics +15