Exon-skipping advances for Duchenne muscular dystrophy
Author:
Affiliation:
1. U1179 INSERM, UFR des Sciences de la Santé, Montigny le Bretonneux, France
2. SQY Therapeutics, Université de Versailles St-Quentin , Montigny le Bretonneux, France
Funder
Agence nationale de la recherche
Association Monegasque contre les myopathies
Duchenne Parent project France
Publisher
Oxford University Press (OUP)
Subject
Genetics(clinical),Genetics,Molecular Biology,General Medicine
Link
http://academic.oup.com/hmg/article-pdf/27/R2/R163/25229914/ddy171.pdf
Reference74 articles.
1. The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review;Ryder;Orphanet. J. Rare Dis,2017
2. The importance of genetic diagnosis for Duchenne muscular dystrophy;Aartsma-Rus;J. Med. Genet,2016
3. The TREAT-NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutations;Bladen;Hum. Mutat,2015
4. Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management;Birnkrant;Lancet Neurol,2018
5. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management;Bushby;Lancet Neurol,2010
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