Muscular Dystrophies clinical trials at UC Irvine

3 in progress, 1 open to eligible people

Showing trials for

  • AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)

    open to eligible people ages 18-65

    The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Participants will be treated in sequential, dose-level cohorts. (Part 1)

    Irvine, California and other locations

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

    Sorry, in progress, not accepting new patients

    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.

    Irvine, California and other locations

  • Defining Endpoints in Becker Muscular Dystrophy

    Sorry, in progress, not accepting new patients

    This is a 24-month, observational study of 50 participants with Becker muscular dystrophy (BMD)

    Orange, California and other locations

Our lead scientists for Muscular Dystrophies research studies include .

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