Pompe disease
SPK-3006-101
Phase 1/2, Dose-escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of SPK-3006 in Adults with Late-onset Pompe Disease The purpose of this...
LSD Registry
Lysosomal Storage Disorders (LSD) Registry Program – Pompe Registry The LSD Registry program is a multi-center, international, observational program for patients with certain rare diseases designed to track...
Lysosomal Storage Disorders (LSD) Registry Program
The Lysosomal Storage Disorders (LSD) Registry Program is a multicenter, international, observational program for people with certain rare diseases. It is designed to track the natural history and...