An open-label ascending dose cohort study to assess the safety, pharmacokinetics, and preliminary efficacy of neoGAA (GZ402666) in patients with infantile-onset Pompe disease treated with alglucosidase alfa who demonstrate clinical decline
Administered By
Awarded By
Contributors
- Kishnani, Priya Sunil Principal Investigator
Start/End
- May 14, 2018 - December 31, 2022