LGMD AUTOSOMAL RESSESSIVE AND DOMINANT

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Disease specific patient reported outcomes measures (PROs) have emerged as clinical devices that are useful to measure disease progression and serve as a validated outcomes for clincal trials. An initial first step is to identify the multitude of symptoms that impact the health and daily function of individuals with limb-girdle muscular dystrophy (LGMD) using a qualitative, patient-directed approach. The LGMDs include over 50 different genetic conditions that result in a shared clinical phenotype.

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