Gangliosidosis

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At a Glance

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Regulatory Watchboard

4 events
Jun 2026A Phase I Study of Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis

Tippi Mackenzie — PHASE1

TrialNOT YET RECRUITING
Jun 2025A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis

Azafaros A.G. — PHASE3

TrialRECRUITING
Aug 2019A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis

National Human Genome Research Institute (NHGRI) — PHASE1, PHASE2

TrialRECRUITING
Dec 2010A Natural History Study of the Gangliosidoses

University of Minnesota

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 Gangliosidosis.
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Clinical Trial Landscape

3 active trials
1Phase 3
1Unknown
1PHASE1, PHASE2
3Total recruiting
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Recent News & Research

No recent news articles indexed yet for Gangliosidosis.
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Specialist Network

Top 6 by expertise

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