GM1 gangliosidosis type 1

MONDO:0009260

GM1 gangliosidosis type 1 is the severe infantile form of GM1 gangliosidosis with variable neurological and systemic manifestations.

Also known as: Norman-Landing disease, infantile GM1 gangliosidosis, Beta galactosidase deficiency type 1, Beta-galactosidase-1 deficiency, GLB deficiency type 1, GM1-gangliosidosis, type 1, GM1-gangliosidosis, type I, GM1-gangliosidosis, type I, with Cardiac involvement

3 clinical trials for this condition and its sub-types, 3 tagged with GM1 gangliosidosis type 1 itself.

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Where it sits in the disease tree

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Part of

↑ Syndromic dyslipidemia (73) ↑ Lysosomal storage disease with skeletal involvement (63) ↑ GM1 gangliosidosis (12)

Including sub-types (3) Tagged with GM1 gangliosidosis type 1 (3)

Trials to join now! 1 Not yet recruiting 1 Not yet finished but already full! 1

Experimental gene therapy offers hope for fatal nerve disease

Disease control Recruiting now

This study tests a gene therapy for GM1 gangliosidosis, a fatal disorder that destroys nerve cells. The therapy uses a harmless virus to deliver a working gene, helping the body produce a missing enzyme. Up to 54 children with Type I (ages 6-12 months) or Type II (ages 1-12 years…

Phase 1/2 • Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Disease control

Last updated Sep 13, 2026 00:00 UTC

GM1 gangliosidosis type 1

Where it sits in the disease tree

Experimental gene therapy offers hope for fatal nerve disease