Glycogen Storage Disease Type II clinical trials at UC Irvine
1 research study open to eligible people
open to eligible people ages 18 years and up
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.