GM1 GANGLIOSIDOSIS
Clinical trials for GM1 GANGLIOSIDOSIS explained in plain language.
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Landmark study maps devastating rare disease in children
Knowledge-focused CompletedThis study aimed to understand how the rare and fatal genetic disorder GM1 gangliosidosis progresses in children. Researchers followed 31 children with the condition for up to 3 years, collecting detailed health information every 6 months. The goal was to create a detailed pictur…
Matched conditions: GM1 GANGLIOSIDOSIS
Sponsor: University of Pennsylvania • Aim: Knowledge-focused
Last updated Apr 01, 2026 14:41 UTC
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Scientists map the progression of rare childhood brain diseases
Knowledge-focused CompletedThis study aimed to understand how neurological symptoms like walking and speech difficulties change over time in children with rare genetic disorders called GM1 or GM2 gangliosidosis. It followed 31 children with late-infantile or juvenile onset of the disease, using doctor asse…
Matched conditions: GM1 GANGLIOSIDOSIS
Sponsor: Azafaros A.G. • Aim: Knowledge-focused
Last updated Mar 27, 2026 12:39 UTC