Landmark study maps devastating rare disease in children
NCT ID NCT04041102
Summary
This study aimed to understand how the rare and fatal genetic disorder GM1 gangliosidosis progresses in children. Researchers followed 31 children with the condition for up to 3 years, collecting detailed health information every 6 months. The goal was to create a detailed picture of the disease's course to help design better future treatment trials.
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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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