LYSOSOMAL STORAGE DISEASES
Clinical trials for LYSOSOMAL STORAGE DISEASES explained in plain language.
Never miss a new study
Get alerted when new LYSOSOMAL STORAGE DISEASES trials appear
Sign up with your email to follow new studies for LYSOSOMAL STORAGE DISEASES, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
By submitting, you agree to our Terms of use
-
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: LYSOSOMAL STORAGE DISEASES
Phase: PHASE1, PHASE2 • Sponsor: University of California, San Diego • Aim: Disease control
Last updated May 15, 2026 11:57 UTC
-
Hunting for hidden genetic clues in rare diseases
Knowledge-focused CompletedThis study looked for new genetic changes that might cause inherited diseases, especially in people who already have a genetic disorder but whose genetic tests were not clear. Researchers examined DNA from 56 participants, including patients and their unaffected family members. T…
Matched conditions: LYSOSOMAL STORAGE DISEASES
Sponsor: Duke University • Aim: Knowledge-focused
Last updated May 11, 2026 20:40 UTC