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Condition: Gangliosidoses Gm2 Synced from ClinicalTrials.gov · August 04, 2026

Recruiting studies: Gangliosidoses Gm2

Studies currently listed as recruiting on the public registry for this search term. Each page explains the study in plain language, with locations and the study team's contact details. Clinicians: generate a printable list for your patient →

Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders
Phase N/A · 1 location(s) · National Human Genome Research Institute (NHGRI) · NCT00029965
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niem
Phase 3 · 34 location(s) · Azafaros B.V. · NCT07082725
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Phase 1 · 1 location(s) · Joanne Kurtzberg, MD · NCT02254863
A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pi
Phase 2 · 3 location(s) · Azafaros B.V. · NCT07399704
A Natural History Study of the Gangliosidoses
Phase N/A · 1 location(s) · University of Minnesota · NCT00668187
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niem
Phase 3 · 38 location(s) · Azafaros B.V. · NCT07054515
Longitudinal Study of Neurodegenerative Disorders
Phase N/A · 1 location(s) · University of Pittsburgh · NCT03333200
The Myelin Disorders Biorepository Project
Phase N/A · 23 location(s) · Children's Hospital of Philadelphia · NCT03047369
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