Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53-Reference-Cited by-同舟云学术

Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53

Published:2019-06-25 Issue:6 Volume:14 Page:e0218683
ISSN:1932-6203
Container-title:PLOS ONE
language:en
Short-container-title:PLoS ONE

Author:

Brogna Claudia,Coratti Giorgia,Pane Marika,Ricotti Valeria,Messina Sonia,D’Amico Adele,Bruno Claudio,Vita Gianluca,Berardinelli Angela,Mazzone Elena,Magri Francesca,Ricci Federica,Mongini Tiziana,Battini Roberta,Bello Luca,Pegoraro Elena,Baranello Giovanni^ORCID,Previtali Stefano C.,Politano Luisa,Comi Giacomo P.,Sansone Valeria A.,Donati Alice,Bertini Enrico^ORCID,Muntoni Francesco,Goemans Nathalie,Mercuri Eugenio^ORCID,

Funder

Fondazione Telethon

Publisher

Public Library of Science (PLoS)

Subject

Multidisciplinary

Reference23 articles.

1. Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study;CM McDonald;Lancet,2018

2. Evidence-based care in Duchenne muscular dystrophy;CM McDonald;Lancet Neurol,2018

3. Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy;JR Mendell;Ann Neurol,2016

4. Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial;CM McDonald;Lancet,2017

5. Non-Ambulant Duchenne Patients Theoretically Treatable by Exon 53 Skipping have Severe Phenotype;L Servais;J Neuromuscul Dis,2015

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