Lysosomal Rare Disorders Research and Treatment Center

Fairfax, Virginia 22030-7404

4 studies enrolling now · 8 studies all time

What they study most

GM1 Gangliosidosis, GM2 Gangliosidosis, Gangliosidoses, GM2, Gangliosidosis, GM1, Gaucher Disease, Gaucher Disease Type 1, Gaucher Disease, Type 1, Gaucher Disease, Type 2, Niemann-Pick Type C Disease

Studies at this site

• A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis — Recruiting now
• A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC) — Recruiting now
• A Gaucher Disease Gene Therapy Trial With FLT201 — Recruiting now
• A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED) — Recruiting now
• A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis — Running, not enrolling
• A Gene Therapy Study in Patients With Gaucher Disease Type 1 — Completed
• Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE) — Running, not enrolling
• Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2) — Running, not enrolling