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 the natural history and outcomes of patients. Currently, patients diagnosed with Pompe disease may participate in this registry program.
No experimental intervention is given; thus, a patient will undergo clinical assessments and receive standard of-care treatment as determined by the patient’s 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