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Limb-Girdle Muscular Dystrophy clinical trials at University of California Health

3 in progress, 2 open to eligible people

Showing trials for
  • A Study of the Natural History of Patients With LGMD2E/R4, LGMD2D/R3, and LGMD2C/R5, ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice

    open to eligible people ages 4 years and up

    This study will follow patients who are screened and confirmed with a genetic diagnosis of Limb-girdle muscular dystrophy type 2E (LGMD2E/R4), Limb-girdle muscular dystrophy type 2D (LGMD2D/R3), or Limb-girdle muscular dystrophy type 2C (LGMD2C/R5). These enrolled patients will be followed to evaluate mobility and pulmonary function for up to 3 years after enrollment. Additional patient data will be collected from the time the individual began experiencing LGMD symptoms to the present.

    at UC Davis

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

    open to eligible people ages 4-65

    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 LGMD2i

    Sorry, in progress, not accepting new patients

    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 LGMD2i to support therapeutic development.

    at UC Irvine

Our lead scientists for Limb-Girdle Muscular Dystrophy research studies include .

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