Great Ormond Street Hospital

London, London WC1N 3JH

2 studies enrolling now · 2 studies all time

What they study most

GM1 Gangliosidosis, GM2 Gangliosidosis, 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