FKRP Mutation clinical trials at UC Irvine

1 in progress, 0 open to eligible people

AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
Sorry, in progress, not accepting new patients
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

Last updated: August 24, 2026