Becker muscular dystrophy (BMD) is an inherited muscle disorder caused by deficiency of dystrophin. The relationship between disease severity and dystrophin levels needs further assessment. In this issue of JNNP, a Dutch group reported an association between dystrophin level s and disease severity in BD. Specifically, dystrophin levels were not a major determinant of disease severity unless dystrophin levels < 10% in muscles. Interestingly the study suggested that disease severity is more dependent on mutation site. So, where to from here, and what should be our treatment strategies?
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- Research paper