Anna Łusakowska

The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations

Catherine L. Bladen, David Salgado, Soledad Monges et al. · Human Mutation · 2015 · 717 citations · Full text

Dmd Mutations, Trial Planning, Engineering +12

A collaborative study on the natural history of childhood and juvenile onset proximal spinal muscular atrophy (type II and III SMA): 569 patients

Klaus Zerres, Sabine Rudnik‐Schöneborn, Eric Forrest et al. · Journal of the Neurological Sciences · 1997 · 282 citations

Neuromusculoskeletal Disorder, Spinal Disorder, Natural History +9

Clinical Outcomes in Duchenne Muscular Dystrophy: A Study of 5345 Patients from the TREAT-NMD DMD Global Database

Zaïda Koeks, Catherine L. Bladen, David Salgado et al. · Journal of Neuromuscular Diseases · 2017 · 171 citations · Full text

Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial

Enrico Bertini, Éric Dessaud, Eugenio Mercuri et al. · The Lancet Neurology · 2017 · 114 citations · Full text

Placebo-controlled Phase 2, Chronic Musculoskeletal Condition, Type 2 +6

Sequential targeted exome sequencing of 1001 patients affected by unexplained limb-girdle weakness

Ana Töpf, Katherine Johnson, Adam Bates et al. · Genetics in Medicine · 2020 · 105 citations · Full text