CYSTINOSIS
Clinical trials for CYSTINOSIS explained in plain language.
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Gene-Fixed stem cells show promise against rare kidney disease
Disease control CompletedThis study tested a gene therapy for cystinosis, a rare disease that causes cystine crystals to build up and damage organs. Researchers took blood stem cells from 6 patients, fixed the faulty gene in a lab, and put the cells back. The goal was to see if it's safe and if it can lo…
Matched conditions: CYSTINOSIS
Phase: PHASE1, PHASE2 • Sponsor: University of California, San Diego • Aim: Disease control
Last updated May 20, 2026 11:56 UTC
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Cystinosis study: does taking medicine as prescribed protect the brain?
Knowledge-focused CompletedThis study followed 65 people with cystinosis for two years to see how closely they took their cysteamine medication and whether that affected their brain health. Researchers used special devices to track medication use, performed memory and vision tests, and used brain scans to …
Matched conditions: CYSTINOSIS
Phase: NA • Sponsor: Hospices Civils de Lyon • Aim: Knowledge-focused
Last updated May 26, 2026 07:45 UTC
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Scientists dig into mysterious skin lightening in 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. They studied the genes and skin of participants to find the link between cystinosis and skin …
Matched conditions: CYSTINOSIS
Phase: NA • Sponsor: Centre Hospitalier Universitaire de Nice • Aim: Knowledge-focused
Last updated May 26, 2026 07:35 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