GM2 gangliosidosis

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At a Glance

Live from database

Regulatory Watchboard

5 events
May 2026Translational Potential of ex Vivo Gene Therapy in GM2 Gangliosidosis

Assistance Publique - Hôpitaux de Paris

TrialNOT YET RECRUITING
Feb 2026A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

Azafaros A.G. — PHASE2

TrialRECRUITING
Sep 2024Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease

Terence Flotte

TrialACTIVE NOT RECRUITING
Apr 2023Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients with GM2 Gangliosidosis or Niemann-Pick Type C Disease

Azafaros A.G. — PHASE2

TrialACTIVE NOT RECRUITING
Sep 2022Caregiving Networks Across Disease Context and the Life Course

National Human Genome Research Institute (NHGRI)

TrialRECRUITING

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

FINANCIAL LANDSCAPE SUMMARY

0

Total programs

No financial assistance programs currently listed for GM2 gangliosidosis.
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Clinical Trial Landscape

4 active trials
2Phase 2
2Unknown
4Total recruiting
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Recent News & Research

No recent news articles indexed yet for GM2 gangliosidosis.
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Specialist Network

Top 6 by expertise

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