CYSTINOSIS
Clinical trials for CYSTINOSIS explained in plain language.
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Scientists test One-Time gene fix for devastating rare disease
Disease control CompletedThis early-stage study tested a one-time gene therapy for cystinosis, a rare and serious genetic disorder that causes cystine to build up and damage organs. Researchers collected stem cells from 6 patients, used a virus to insert a corrected gene into those cells, and then infuse…
Matched conditions: CYSTINOSIS
Phase: PHASE1, PHASE2 • Sponsor: University of California, San Diego • Aim: Disease control
Last updated Apr 01, 2026 14:43 UTC
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Scientists probe why cystinosis treatment fails to protect the brain
Knowledge-focused CompletedThis study aimed to understand how consistently patients with cystinosis take their cysteamine medication and how that affects cystine levels in their bodies and potential brain complications. Researchers followed 65 patients over two years, using electronic monitors to track pil…
Matched conditions: CYSTINOSIS
Phase: NA • Sponsor: Hospices Civils de Lyon • Aim: Knowledge-focused
Last updated Apr 02, 2026 00:41 UTC
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Doctors map hidden toll of rare disease on adult bodies
Knowledge-focused CompletedThis study aimed to understand how cystinosis, a rare genetic disorder, affects muscle strength, breathing, and swallowing in older teens and adults. Researchers followed 20 patients for one year, performing detailed physical tests to track changes in their abilities. The goal wa…
Matched conditions: CYSTINOSIS
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Mar 27, 2026 12:39 UTC