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Limb-Girdle Muscular Dystrophy clinical trials at UC Health
2 in progress, 1 open to eligible people

  • Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD)

    open to eligible people ages 6-65

    Limb Girdle Muscular Dystrophy comprise a group of disorders made up of over 30 mutations which share a common phenotype of progressive weakness of the shoulder and hip girdle muscles. While the individual genetic mutations are rare, as a cohort, LGMDs are one of the four most common muscular dystrophies. The overall goal of project 1 is to define the key phenotypes as measured by standard clinical outcome assessments (COAs) for limb girdle muscular dystrophies (LGMD) to hasten therapeutic development.

    at UC Irvine

  • Biomarker Development in LGMD 2i

    Sorry, accepting new patients by invitation only

    The overall goal of this natural history study is to define the key LGMD2i phenotypes as measured by standard clinical outcome assessments (COAs), and to validate a muscle biomarker for LGMD 2i to support therapeutic development.

    at UC Irvine

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