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 outcomes of patients. Currently, patients diagnosed with Pompe disease may participate in this registry program.
No experimental intervention is given as part of this program. As such, participants will be assessed clinically and receive standard-of-care treatment as determined by their physician. Physicians will determine the actual frequency of necessary assessments according to a patient’s individualized need for medical care and routine follow up.
Principal Investigator: Shafeeq Ladha, MD