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Natural history of LGMD2A for delineating outcome measures in clinical trials

Natural history of LGMD2A for delineating outcome measures in clinical trials

https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4818744

Natural history of LGMD2A for delineating outcome measures in clinical trials

About this item

Full title

Natural history of LGMD2A for delineating outcome measures in clinical trials

Publisher

United States: Wiley

Journal title

Annals of clinical and translational neurology, 2016-04, Vol.3 (4), p.248-265

Language

English

Formats

Publication information

Publisher

United States: Wiley

More information

Scope and Contents

Contents

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 progression in genetically confirmed LGMD2A patie...

Alternative Titles

Full title

Natural history of LGMD2A for delineating outcome measures in clinical trials

Identifiers

Primary Identifiers

Record Identifier

TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4818744

Permalink

https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4818744

Other Identifiers

ISSN

2328-9503

E-ISSN

2328-9503

DOI

10.1002/acn3.287

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