CYSTINOSIS
Clinical trials for CYSTINOSIS explained in plain language.
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Cystinosis drug under Long-Term watch: could cysteamine be key?
Disease control Recruiting nowCystinosis is a rare inherited disease that causes kidney failure and poor growth due to cystine buildup in cells. This study follows 330 patients taking cysteamine, a drug that lowers cystine levels, to track long-term effects, detect new complications, and support genetic resea…
Matched conditions: CYSTINOSIS
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Disease control
Last updated Jul 02, 2026 00:00 UTC
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Rare disease database aims to boost cystinosis care across europe
Knowledge-focused Recruiting nowThis European study will follow 400 people with cystinosis, a rare genetic disease that causes cystine buildup in cells. Researchers will collect medical and quality-of-life data to understand how the disease progresses and how care can be improved. The study does not test any ne…
Matched conditions: CYSTINOSIS
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:04 UTC