GM1 GANGLIOSIDOSIS
Clinical trials for GM1 GANGLIOSIDOSIS explained in plain language.
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Rare disease study maps how children's movement and speech change over time
Knowledge-focused CompletedThis study followed 31 children with late-infantile or juvenile GM1 or GM2 gangliosidosis (including Tay-Sachs and Sandhoff disease) to understand how their neurological symptoms progress. Researchers measured changes in walking and speech using a standard rating scale. The goal …
Matched conditions: GM1 GANGLIOSIDOSIS
Sponsor: Azafaros A.G. • Aim: Knowledge-focused
Last updated May 17, 2026 12:57 UTC
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Tracking a rare disease: study maps GM1's course to speed future cures
Knowledge-focused CompletedThis study followed 31 children with GM1 gangliosidosis, a rare and severe genetic disorder, for up to 3 years. Researchers collected data from exams, blood tests, and imaging to understand how the disease progresses. No treatment was given; the goal was to identify useful marker…
Matched conditions: GM1 GANGLIOSIDOSIS
Sponsor: University of Pennsylvania • Aim: Knowledge-focused
Last updated May 14, 2026 12:05 UTC