FKRP Mutation clinical trials at UC Irvine
1 research study open to eligible people
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 LION-101 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
Our lead scientists for FKRP Mutation research studies include Tahseen Mozaffar, MD.