Rare disease study tracks GM1 progression to pave way for future treatments
NCT ID NCT04041102
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This 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 identify useful markers for future clinical trials, not to test a treatment.
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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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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