GM1 gangliosidosis
MONDO:0018149A rare lysosomal storage disorder characterized biochemically by deficient beta-galactosidase activity and clinically by a wide range of variable neurovisceral, ophthalmological and dysmorphic features.
Also known as: Beta-galactosidase-1 deficiency, GLB1 deficiency, GM>1< gangliosidosis, Landing disease, Landing syndrome, gangliosidosis GM1, Beta galactosidase 1 deficiency, Beta-galactosidosis
12 clinical trials for this condition and its sub-types.
Follow this condition — get notified about new trialsSub-types
Broader categories
-
Rare disease study tracks GM1 progression to pave way for future treatments
Knowledge-focused CompletedThis study followed 31 children with infantile or juvenile GM1 gangliosidosis, a rare and severe genetic disorder, for up to 3 years. Researchers collected data from exams, blood tests, and developmental assessments to better understand how the disease progresses. The goal was to…
Sponsor: University of Pennsylvania • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:55 UTC
-
New study maps how rare brain diseases worsen over time in kids
Knowledge-focused CompletedThis study followed 31 children with GM1 or GM2 gangliosidosis (including Tay-Sachs and Sandhoff disease) to carefully measure how their neurological symptoms, like walking and speech, change over time. The goal was to create a clear picture of disease progression to help design …
Sponsor: Azafaros B.V. • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:52 UTC