CYSTINOSIS
Clinical trials for CYSTINOSIS explained in plain language.
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Gene therapy trial offers new hope for cystinosis patients
Disease control CompletedThis study tested a new gene therapy for cystinosis, a rare genetic disease that causes harmful crystals to build up in cells. Researchers took stem cells from 6 patients, corrected the faulty gene in a lab, and gave the cells back. The goal was to see if this approach is safe an…
Matched conditions: CYSTINOSIS
Phase: PHASE1, PHASE2 • Sponsor: University of California, San Diego • Aim: Disease control
Last updated May 15, 2026 11:57 UTC
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Scientists dig into skin clues for rare disease
Knowledge-focused CompletedThis study looked at 30 people with cystinosis, a rare disease that causes crystals to build up in the body. Researchers wanted to understand why some patients have lighter skin patches and how that relates to the disease. The goal was to learn more about the disease's biology, n…
Matched conditions: CYSTINOSIS
Phase: NA • Sponsor: Centre Hospitalier Universitaire de Nice • Aim: Knowledge-focused
Last updated May 17, 2026 07:03 UTC
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New study maps muscle decline in rare cystinosis patients
Knowledge-focused CompletedThis study looked at how cystinosis affects muscles and nerves in 20 patients aged 16 and older. Researchers measured motor function, breathing, swallowing, and sleep over time. The goal was to better understand the disease's progression, not to test a new treatment.
Matched conditions: CYSTINOSIS
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated May 14, 2026 12:06 UTC
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Cystinosis study probes drug compliance and brain risks
Knowledge-focused CompletedThis study looked at 65 people with cystinosis to see how well they take their cysteamine medicine and if that affects brain problems. Researchers measured drug use with special bottle caps and diaries, and used brain scans and memory tests to check for cystine buildup. The goal …
Matched conditions: CYSTINOSIS
Phase: NA • Sponsor: Hospices Civils de Lyon • Aim: Knowledge-focused
Last updated May 14, 2026 12:03 UTC