Advances in congenital myopathies

Congenital myopathies are a heterogenous group of rare diseases characterised by structural abnormalities in muscle fibres which are most often detected at a young age. These abnormalities lead to muscle weakness (hypotonia and impaired motor skills) which generally manifests at birth or during the first few months of life (“congenital”).

This document, presents research news on congenital myopathies from the past year (ongoing studies and clinical trials, scientific and medical publications, etc.).


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