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Article

Natural history of limb girdle muscular dystrophy R9 over 6 years: searching for trial endpoints

16 May 2019

Abstract excerpt

OBJECTIVE: Limb girdle muscular dystrophy type R9 (LGMD R9) is an autosomal recessive muscle disease for which there is currently no causative treatment. The development of putative therapies requires sensitive outcome measures for clinical trials in this slowly progressing condition. This study extends functional assessments and MRI muscle fat fraction measurements in an LGMD R9 cohort across 6 years. METHODS:...

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PMID: 31211167
DOI: 10.1002/acn3.774

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