Scientists map the progression of rare childhood brain diseases
NCT ID NCT05109793
Summary
This 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 assessments and parent questionnaires, often through virtual visits. The goal was to gather detailed information to help future research, not to test a treatment.
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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
Explore the condition pages connected to this study.