Autosomal recessive inheritance of RYR1 mutations in a congenital myopathy with cores.

Jungbluth, H; Müller, C R; Halliger-Keller, B; Brockington, M; Brown, S C; Feng, L; Chattopadhyay, A; Mercuri, E et al. · Neurology · 2002

case_series · Level IV

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Abstract

Central core disease (CCD) is a congenital myopathy due to dominant mutations in the skeletal muscle ryanodine receptor gene (RYR1). The authors report three patients from two consanguineous families with symptoms of a congenital myopathy, cores on muscle biopsy, and confirmed linkage to the RYR1 locus. Molecular genetic studies in one family identified a V4849I homozygous missense mutation in the RYR1 gene. This report suggests a congenital myopathy associated with recessive RYR1 mutations.

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