Tracking a rare disease: study maps GM1's course to speed future cures
NCT ID NCT04041102
First seen Nov 01, 2025 · Last updated May 14, 2026 · Updated 26 times
Summary
This 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 markers for future clinical trials.
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the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Gazi University
Ankara, Turkey (Türkiye)
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Hospital de Clínicas de Porto Alegre
Porto Alegre, Brazil
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Montreal Children's Hospital Research Institute - McGill University
Montreal, Quebec, Canada
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UCL Great Ormond Street Institute of Child Health
London, United Kingdom
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UCSF Benioff Children's Hospital Oakland
Oakland, California, 94610, United States
Conditions
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