Rare disease study maps muscle decline in cystinosis patients
NCT ID NCT05545774
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study looked at how cystinosis affects muscles, breathing, and swallowing in 20 teenagers and adults. Researchers measured motor function, strength, and walking ability over time. The goal was to better understand the disease's impact on the body, not to test a new treatment.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Newborn screening study aims to catch rare diseases at birth
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- Rare disease database aims to boost cystinosis care across europe
- Experimental gene therapy aims to fix cystinosis at the source
- Cystinosis study probes medication adherence and brain risks