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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Detalles Bibliográficos
Autores principales: Walter, Maggie C, Reilich, Peter, Thiele, Simone, Schessl, Joachim, Schreiber, Herbert, Reiners, Karlheinz, Kress, Wolfram, Müller-Reible, Clemens, Vorgerd, Matthias, Urban, Peter, Schrank, Bertold, Deschauer, Marcus, Schlotter-Weigel, Beate, Kohnen, Ralf, Lochmüller, Hanns
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2013
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3617000/
https://www.ncbi.nlm.nih.gov/pubmed/23406536
http://dx.doi.org/10.1186/1750-1172-8-26

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