Hope for cystinosis: drug may slow damage, but cure still elusive
NCT ID NCT00359684
First seen Jan 07, 2026 · Last updated May 01, 2026 · Updated 19 times
Summary
This study follows 330 people with cystinosis, a rare inherited disease that causes kidney failure and growth problems. The drug cysteamine helps lower harmful cystine buildup in cells. Researchers aim to track long-term effects, find new complications, and support genetic testing.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
Conditions
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