Muscular Dystrophies clinical trials at UC Irvine
3 in progress, 1 open to eligible people
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
Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping
Sorry, not accepting new patients
The purpose of this Managed Access Program is to allow access to delpacibart zotadirsen (AOC 1044) for eligible patients diagnosed with DMD mutations amenable to exon 44 skipping. The patient's Administering Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.
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 Tahseen Mozaffar, MD.
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