Abstract
Becker muscular dystrophy features progressive proximal weakness, wasting and often focal hypertrophy. We present a patient with pain and cramps from adolescence. Widespread muscle hypertrophy, preserved muscle strength and a 10-20-fold raised CPK were noted. Muscle biopsy was dystrophic, and Western blot showed a 95% reduction of dystrophin levels. Genetic analyses revealed a non-sense mutation in exon 2 of the dystrophin gene. This mutation is predicted to result in a Duchenne phenotype, but resulted in a mild Becker muscular dystrophy with widespread muscle hypertrophy. We suggest that this unusual phenotype is caused by translation re-initiation downstream from the mutation site.
Originalsprog | Engelsk |
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Tidsskrift | Neuromuscular Disorders |
Vol/bind | 23 |
Udgave nummer | 1 |
Sider (fra-til) | 25-8 |
Antal sider | 4 |
ISSN | 0960-8966 |
DOI | |
Status | Udgivet - 2013 |