Report on the workshop: Meaningful outcome measures for Duchenne muscular dystrophy, London, UK, 30

Report on the workshop: Meaningful outcome measures for Duchenne muscular dystrophy, London, UK, 30–31 January 2017

Published:2018-08 Issue:8 Volume:28 Page:690-701
ISSN:0960-8966
Container-title:Neuromuscular Disorders
language:en
Short-container-title:Neuromuscular Disorders

Author:

Straub Volker^ORCID,Mercuri Eugenio,Aartsma-Rus Annemieke,Athanasiou Dimitrios,Balabanov Pavel,Buccella Filippo,Caizergues Didier,Carlier Pierre,Crossley Emily,Connolly Anne M.,Duong Tina,Fischer Dirk,Furlong Pat,Goemans Nathalie,de Groot Imelda,Guglieri Michela,Henricson Erik,Johnson Alex,Kan Hermien E.,Mayhew Anna,Mazzone Elena,McDonald Craig M.,Mercuri Eugenio,Muntoni Francesco,Niks Erik H.,Servais Laurent,Turner Cathy,Voit Thomas,Vroom Elizabeth,Walter Glenn

Publisher

Elsevier BV

Subject

Genetics(clinical),Clinical Neurology,Neurology,Pediatrics, Perinatology, and Child Health

Reference43 articles.

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

2. European medicines agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene;Haas;Neuromuscul Disord,2016

3. FDA approves Eteplirsen for Duchenne muscular dystrophy: the next chapter in the Eteplirsen saga;Aartsma-Rus;Nucleic Acid Ther,2017

4. Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy;Straub;Lancet Neurol,2016

5. Development of exon skipping therapies for Duchenne muscular dystrophy: a critical review and a perspective on the outstanding issues;Aartsma-Rus;Nucleic Acid Ther,2017

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