Lysosomal and Rare Disorders Research and Treatment Center

Fairfax, Virginia 22030

1 studies enrolling now · 5 studies all time

What they study most

AB Variant Gangliosidosis GM2, Fabry Disease, Fabry Disesase, GM1 Gangliosidosis, GM2 Gangliosidosis, Gaucher Disease, Gaucher Disease, Type 2, Lysosomal Storage Diseases, Sandhoff Disease, Tay-Sachs Disease

Studies at this site

• A proof-of Concept Study to Assess Safety and Tolerability of HM15421/GC1134A in Patients With Fabry Disease — Recruiting now
• A Fabry Disease Gene Therapy Study — Stopped early
• Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease — Completed
• Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease — Completed
• Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2 — Completed