Glycogen Storage Disease Type II clinical trials at UC Irvine
1 in progress, 0 open to eligible people
Sorry, currently not accepting new patients, but might later
The purpose of this study is to evaluate the safety, tolerability, and efficacy of a single intravenous infusion of SPK-3006 in adults with clinically moderate, late-onset Pompe disease receiving enzyme replacement therapy (ERT). Participants will be treated in sequential, dose-level cohorts.