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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Contacts and locations

Locations

  • Armand-Trousseau Children's Hospital - CHU Paris Est

    Paris, France

  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    Milan, Italy

  • Great Ormond Street Hospital NHSFT

    London, United Kingdom

  • Hopital d'Enfants CHU Timone

    Marseille, France

  • Hospital Pequeno Principe

    Curitiba, Brazil

  • Hospital de Clinicas de Porto Alegre

    Porto Alegre, Brazil

  • Hôpital des Enfants - CHU Toulouse Purpan

    Toulouse, France

  • LMU - Klinikum der Universitaet Muenchen - Neurologische Klinik und Poliklinik

    Munich, Germany

  • Mayo Clinic Rochester

    Rochester, Minnesota, 55905, United States

  • UCSF Benioff Children's Hospital

    Oakland, California, 94609, United States

  • Universita' di Catania

    Catania, Italy

  • University Hospital Friuli Centrale

    Udine, Italy

  • Universtitäsklinikum Giessen und Marburg

    Giessen, Germany

Conditions

Explore the condition pages connected to this study.