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DMD genotypes and loss of ambulation in the CINRG Duchenne Natural History Study

OBJECTIVE: To correlate time to loss of ambulation (LoA) and different truncating DMD gene mutations in a large, prospective natural history study of Duchenne muscular dystrophy (DMD), with particular attention to mutations amenable to emerging molecular treatments. METHODS: We analyzed data from th...

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Detalhes bibliográficos
Publicado no:Neurology
Main Authors: Bello, Luca, Morgenroth, Lauren P., Gordish-Dressman, Heather, Hoffman, Eric P., McDonald, Craig M., Cirak, Sebahattin
Formato: Artigo
Idioma:Inglês
Publicado em: Lippincott Williams & Wilkins 2016
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4977110/
https://ncbi.nlm.nih.gov/pubmed/27343068
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1212/WNL.0000000000002891
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