Scientists map the hidden toll of cystinosis on the body
NCT ID NCT05545774
Summary
This study aimed to understand how cystinosis, a rare genetic disease, affects muscle strength, walking, and breathing in patients aged 16 and older. Researchers followed 20 participants for a year, measuring changes in their physical abilities. The goal was to gather detailed information to better manage the disease's long-term effects, not to test a new treatment.
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Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
Locations
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Department of Nephrology and Transplantation Centre de référence des Maladies Rénales Héréditaires de l'Enfant et de l'Adulte, Necker-Enfants Malades University Hospital, APHP
Paris, France
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Neurology Department, Raymond-Poincaré hospital, APHP
Garches, 92380, France
Conditions
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