Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)

Fairfax, Virginia 22030

2 studies enrolling now · 3 studies all time

What they study most

Fabry Disease, Mucopolysaccharidosis IIIB, Pompe Disease

Studies at this site

• Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD) — Recruiting now
• A Phase I/II Trial of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB) — Starting soon
• Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR) — Completed