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Treatment of dysferlinopathy with deflazacort: a double-blind, placebo-controlled clinical trial
BACKGROUND: Dysferlinopathies are autosomal recessive disorders caused by mutations in the dysferlin (DYSF) gene encoding the dysferlin protein. DYSF mutations lead to a wide range of muscular phenotypes, with the most prominent being Miyoshi myopathy (MM) and limb girdle muscular dystrophy type 2B...
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Main Authors: | , , , , , , , , , , , , , , |
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Formato: | Artigo |
Idioma: | Inglês |
Publicado em: |
BioMed Central
2013
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Assuntos: | |
Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3617000/ https://ncbi.nlm.nih.gov/pubmed/23406536 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1750-1172-8-26 |
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