A mini-review and implementation model for using ataluren to treat nonsense mutation Duchenne muscular dystrophy-Reference-Cited by-同舟云学术

A mini-review and implementation model for using ataluren to treat nonsense mutation Duchenne muscular dystrophy

Published:2018-10-02 Issue:2 Volume:108 Page:224-230
ISSN:0803-5253
Container-title:Acta Paediatrica
language:en
Short-container-title:Acta Paediatr

Author:

Landfeldt Erik¹²^ORCID,Sejersen Thomas³,Tulinius Már⁴

Affiliation:

1. Institute of Environmental Medicine; Karolinska Institutet; Stockholm Sweden

2. ICON plc; Stockholm Sweden

3. The Department of Women's and Children's Health, Paediatric Neurology; Karolinska University Hospital; Astrid Lindgren Children's Hospital; Karolinska Institutet; Stockholm Sweden

4. Department of Pediatrics; Queen Silvia Children's Hospital; University of Gothenburg; Gothenburg Sweden

Publisher

Wiley

Subject

General Medicine,Pediatrics, Perinatology, and Child Health

Link

http://onlinelibrary.wiley.com/wol1/doi/10.1111/apa.14568/fullpdf

Reference36 articles.

1. The muscular dystrophies;Emery;Lancet,2002

2. Origins and early descriptions of “Duchenne muscular dystrophy”;Tyler;Muscle Nerve,2003

3. The medical genetics of dystrophinopathies: molecular genetic diagnosis and its impact on clinical practice;Ferlini;Neuromuscul Disord,2013

4. Current status of pharmaceutical and genetic therapeutic approaches to treat DMD;Pichavant;Mol Ther,2011

5. The National Institute for Health and Care Excellence NICE guidance for ataluren https://www.nice.org.uk/guidance

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