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WMS 2020

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Genotype-phenotype associations in people with limb girdle muscular dystrophy with sarcoglycanopathy

Takeaway

  • Onset of symptoms before 10 years of age, in people with limb girdle muscular dystrophy (LGMD), was associated with losing the ability to walk before adulthood.

Why this matters

  • The findings can be used to support the design of future clinical trials and gene replacement therapies, in order to improve the quality of life of those with LGMD.

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