Natural history of LGMD 2A for delineating outcome measures in clinical trials

Richard, Isabelle; Hogrel, Jean‐Yves; Stockholm, Daniel; Payan, Christine; Fougerousse, Françoise; Calpainopathy Study Group, The; Eymard, B.; Mignard, Claude; López de Munaín, Adolfo; Fardeau, Michel; Andoni Urtizberea, Jon

doi:10.1002/acn3.287

Public

Natural history of LGMD 2A for delineating outcome measures in clinical trials

Published in Annals of Clinical and Translational Neurology • Mar 4, 2016

Authors:

Isabelle Richard

Jean‐Yves Hogrel

Daniel Stockholm

Abstract

OBJECTIVE: Limb-girdle muscular dystophy 2A (LGMD2A, OMIM) is a slowly progressive myopathy caused by the deficiency in calpain 3, a calcium-dependent cysteine protease of the skeletal muscle. METHODS: In this study, we carried out an observational study of clinical manifestations and disease progre...

Subject

Medicine

Muscle weakness

Weakness

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