Non-Ambulant Duchenne Patients Theoretically Treatable by Exon 53 Skipping have Severe Phenotype-Reference-Cited by-同舟云学术

Non-Ambulant Duchenne Patients Theoretically Treatable by Exon 53 Skipping have Severe Phenotype

Published:2015-09-02 Issue:3 Volume:2 Page:269-279
ISSN:2214-3599
Container-title:Journal of Neuromuscular Diseases
language:
Short-container-title:JND

Author:

Servais Laurent¹,Montus Marie²,Guiner Caroline Le²³,Ben Yaou Rabah¹⁴,Annoussamy Mélanie¹,Moraux Amélie¹,Hogrel Jean-Yves¹,Seferian Andreea M.¹,Zehrouni Karima¹,Le Moing Anne-Gaëlle¹,Gidaro Teresa¹,Vanhulle Catherine⁵,Laugel Vincent⁶,Butoianu Nina⁷,Cuisset Jean-Marie⁸,Sabouraud Pascal⁹,Cances Claude¹⁰,Klein Andrea¹¹,Leturcq France¹⁴,Moullier Philippe²³,Voit Thomas¹

Affiliation:

1. Institut de Myologie, Groupe hospitalier La Pitié Salpêtrière, AP-HP, Paris, France

2. Généthon, Evry, France

3. Atlantic Gene Therapies, INSERM UMR, Nantes, France

4. Laboratoire de biochimie et génétique moléculaire, Hôpital Cochin, APHP, Paris, France

5. Service de Pédiatrie, Centre Hospitalier Universitaire (CHU), Rouen, France

6. Service de Pédiatrie, Centre Hospitalier Universitaire (CHU), Strasbourg, France

7. Pediatric Neurology Clinic, Alexandru Obregia Hospital, Bucharest, Romania

8. Service de Neuropédiatrie, Centre hospitalier régional universitaire et faculté de médecine, Lille, France

9. Service de Pédiatrie A, Hôpital Américain, CHU de Reims, Reims, France

10. Service de Neurologie Pédiatrique, Hôpital des Enfants, Toulouse, France

11. Department of pediatric neurology, University Children’s Hospital, Zurich, Switzerland

Publisher

IOS Press

Subject

Neurology (clinical),Neurology

Reference29 articles.

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3. Systemic administration of PRO051 in Duchenne’s muscular dystrophy;Goemans;The New England Journal of Medicine,2011

4. Therapeutic approaches to muscular dystrophy;Goyenvalle;Hum Mol Genet,2011

5. New therapies for Duchenne muscular dystrophy: Challenges, prospects and clinical trials;Cossu;Trends Mol Med,2007

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