Researchers in NYU Langone’s Division of Neurogenetics conduct clinical trials to advance the understanding and treatment of lysosomal storage disorders and other genetic disorders affecting the nervous system.

Cerliponase Alfa Observational Study
Principal investigator: Nicolas J. Abreu, MD

Gaucher Disease Outcome Survey (GOS)
Principal investigator: Nicolas J. Abreu, MD

Genzyme LSD Registry (Gaucher)
Principal investigator: Nicolas J. Abreu, MD

Genzyme LSD Registry (Pompe)
Principal investigator: Nicolas J. Abreu, MD

Genzyme LSD Registry (Fabry)
Principal investigator: Nicolas J. Abreu, MD

NPC Early Access Program
Principal investigator: Nicolas J. Abreu, MD

A Safety and Efficacy Study of N-Acetyl-L-Leucine on GM2 Gangliosidosis
Principal investigator: Claire Miller, MD, PhD

An Open-label Study of Intrathecal SHP611 in Subjects with Metachromatic Leukodystrophy
Principal investigator: Devorah Segal, MD, PhD

Long-Term Follow-Up of Subjects Who Received SB-318, SB-913, or SB-FIX
Principal investigator: Nicolas J. Abreu, MD

A Study of Soticlestat in Adults and Children with Rare Epilepsies
Principal investigator: Orrin Devinsky, MD

A Study to Assess the Long-Term Safety and Efficacy of ATB200/AT2221 in Adult Subjects with LOPDi19-01680
Principal Investigator: Nicolas J. Abreu, MD