Natural history of <scp>LGMD</scp>2A for delineating outcome measures in clinical trials

Richard, Isabelle; Hogrel, Jean‐Yves; Stockholm, Daniel; A. M. Payan, Christine; Fougerousse, Françoise; Calpainopathy Study Group, The; Eymard, Bruno; Mignard, Claude; Lopez de Munain, Adolfo; Fardeau, Michel; Andoni Urtizberea, Jon

doi:10.1002/acn3.287

Public

Natural history of LGMD2A for delineating outcome measures in clinical trials

Shared by NobleBlocks on Mar 4, 2016 • 12:00 AM UTC

Authors:

Isabelle Richard

Jean‐Yves Hogrel

Daniel Stockholm

Abstract

Abstract Objective Limb‐girdle muscular dystophy 2A ( LGMD 2A, 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 dise...

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