Expanding the phenotype of GMPPB mutations

M Cabrera-Serrano, R Ghaoui, G Ravenscroft… - Brain, 2015 - academic.oup.com
M Cabrera-Serrano, R Ghaoui, G Ravenscroft, RD Johnsen, MR Davis, A Corbett, S Reddel…
Brain, 2015academic.oup.com
Dystroglycanopathies are a heterogeneous group of diseases with a broad phenotypic
spectrum ranging from severe disorders with congenital muscle weakness, eye and brain
structural abnormalities and intellectual delay to adult-onset limb-girdle muscular
dystrophies without mental retardation. Most frequently the disease onset is congenital or
during childhood. The exception is FKRP mutations, in which adult onset is a common
presentation. Here we report eight patients from five non-consanguineous families where …
Abstract
Dystroglycanopathies are a heterogeneous group of diseases with a broad phenotypic spectrum ranging from severe disorders with congenital muscle weakness, eye and brain structural abnormalities and intellectual delay to adult-onset limb-girdle muscular dystrophies without mental retardation. Most frequently the disease onset is congenital or during childhood. The exception is FKRP mutations, in which adult onset is a common presentation. Here we report eight patients from five non-consanguineous families where next generation sequencing identified mutations in the GMPPB gene. Six patients presented as an adult or adolescent-onset limb-girdle muscular dystrophy, one presented with isolated episodes of rhabdomyolysis, and one as a congenital muscular dystrophy. This report expands the phenotypic spectrum of GMPPB mutations to include limb-girdle muscular dystrophies with adult onset with or without intellectual disability, or isolated rhabdomyolysis.
Oxford University Press