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Article

Dystrophin levels and clinical severity in Becker muscular dystrophy patients

29 Nov 2013

Abstract excerpt

OBJECTIVE: Becker muscular dystrophy (BMD) is characterised by broad clinical variability. Ongoing studies exploring dystrophin restoration in Duchenne muscular dystrophy ask for better understanding of the relation between dystrophin levels and disease severity. We studied this relation in BMD patients with varying mutations, including a large subset with an exon 45-47 deletion. METHODS: Dystrophin was...

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PMID: 24292997
DOI: 10.1136/jnnp-2013-306350

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