CYSTINOSIS
Clinical trials for CYSTINOSIS explained in plain language.
Never miss a new study
Get alerted when new CYSTINOSIS trials appear
Sign up with your email to follow new studies for CYSTINOSIS, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
Genom att skicka in godkänner du våra Användarvillkor
-
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
-
Scientists map the hidden toll of cystinosis on the body
Knowledge-focused CompletedThis study aimed to understand how cystinosis, a rare genetic disease, affects muscle strength, walking, and breathing in patients aged 16 and older. Researchers followed 20 participants for a year, measuring changes in their physical abilities. The goal was to gather detailed in…
Matched conditions: CYSTINOSIS
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Apr 03, 2026 14:43 UTC
-
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 14:56 UTC