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RNA Biology|April 7, 2022
Circulating small RNA signatures differentiate accurately the subtypes of muscular dystrophies: small-RNA next-generation sequencing analytics and functional insightsAndrea C Kakouri, Demetris Koutalianos, Andrie Koutsoulidou, et al.The Lancet. Neurology|May 3, 2017
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 trialEnrico Bertini, Eric Dessaud, Eugenio Mercuri, et al.Molecular Therapy. Methods & Clinical Development|October 27, 2021
miR-223-3p and miR-24-3p as novel serum-based biomarkers for myotonic dystrophy type 1Demetris Koutalianos, Andrie Koutsoulidou, Chrystalla Mytidou, et al.Journal of Neuromuscular Diseases|December 3, 2016
European Cross-Sectional Survey of Current Care Practices for Duchenne Muscular Dystrophy Reveals Regional and Age-Dependent DifferencesJulia Vry, Kathrin Gramsch, Sunil Rodger, et al.Orphanet Journal of Rare Diseases|September 7, 2018
Eight years after an international workshop on myotonic dystrophy patient registries: case study of a global collaboration for a rare diseaseLibby Wood, Guillaume Bassez, Corinne Bleyenheuft, et al.Orphanet Journal of Rare Diseases|August 17, 2019
Correction to: Eight years after an international workshop on myotonic dystrophy patient registries: case study of a global collaboration for a rare diseaseLibby Wood, Guillaume Bassez, Corinne Bleyenheuft, et al.Neurology. Genetics|December 30, 2020
Ethnicity-related DMD Genotype Landscapes in European and Non-European CountriesRita Selvatici, Rachele Rossi, Fernanda Fortunato, et al.Journal of Neurology|October 29, 2013
Mapping the differences in care for 5,000 spinal muscular atrophy patients, a survey of 24 national registries in North America, Australasia and EuropeCatherine L Bladen, Rachel Thompson, Jacqueline M Jackson, et al.Human Mutation|January 22, 2015
The TREAT-NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutationsCatherine L Bladen, David Salgado, Soledad Monges, et al.Journal of Neuromuscular Diseases|November 11, 2017
Clinical Outcomes in Duchenne Muscular Dystrophy: A Study of 5345 Patients from the TREAT-NMD DMD Global DatabaseZaïda Koeks, Catherine L Bladen, David Salgado, et al.Pageof 4