Rare disease study maps how children's movement and speech change over time
NCT ID NCT05109793
First seen Nov 01, 2025 · Last updated May 16, 2026 · Updated 26 times
Summary
This 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 was to gather high-quality data on disease trajectory, not to test a treatment.
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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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Armand-Trousseau Children's Hospital - CHU Paris Est
Paris, France
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Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, Italy
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Great Ormond Street Hospital NHSFT
London, United Kingdom
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Hopital d'Enfants CHU Timone
Marseille, France
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Hospital Pequeno Principe
Curitiba, Brazil
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Hospital de Clinicas de Porto Alegre
Porto Alegre, Brazil
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Hôpital des Enfants - CHU Toulouse Purpan
Toulouse, France
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LMU - Klinikum der Universitaet Muenchen - Neurologische Klinik und Poliklinik
Munich, Germany
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Mayo Clinic Rochester
Rochester, Minnesota, 55905, United States
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UCSF Benioff Children's Hospital
Oakland, California, 94609, United States
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Universita' di Catania
Catania, Italy
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University Hospital Friuli Centrale
Udine, Italy
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Universtitäsklinikum Giessen und Marburg
Giessen, Germany
Conditions
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