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.
Genom att skicka in godkänner du våra Användarvillkor
-
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: LYSOSOMAL STORAGE DISEASES
Phase: PHASE1, PHASE2 • Sponsor: University of California, San Diego • Aim: Disease control
Last updated May 20, 2026 11:56 UTC
-
Scientists hunt for missing genetic clues in rare diseases
Knowledge-focused CompletedThis study looked for new or overlooked genetic changes in people with inherited diseases, especially those whose standard genetic tests were unclear. Researchers examined DNA from 56 patients and their family members to find hidden variants that might explain their condition. Th…
Matched conditions: LYSOSOMAL STORAGE DISEASES
Sponsor: Duke University • Aim: Knowledge-focused
Last updated May 22, 2026 14:04 UTC