Recruiting A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD) NCT07282847 Conditions Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more Locations Barrow Neurological Institute — Phoenix, Arizona, United States University of California, Irvine (UCI) — Irvine, California, United States Stanford Neuroscience Health Center — Palo Alto, California, United States NYU Langone — New York, United States View more locations Show fewer locations Loading locations... View Trial Details
Completed Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies. NCT00765414 Conditions Glycogen Storage Disease Type II GSD II, Pompe Disease Late-Onset Locations University of Texas Health Science Center at San Antonio — San Antonio, Texas, United States View Trial Details
Active, Not Recruiting Safety and Efficacy of Avalglucosidase Alfa in Patients With Non-classic Pompe Disease Aged ≥ 5 Years NCT07652814 Conditions Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +17 more Locations Erasmus MC — Rotterdam, Netherlands View Trial Details
