LYSOSOMAL STORAGE DISEASES
Clinical trials for LYSOSOMAL STORAGE DISEASES 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: LYSOSOMAL STORAGE DISEASES
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 hunt for hidden genetic clues in rare diseases
Knowledge-focused CompletedThis study aimed to discover new genetic changes that cause inherited disorders, especially in patients where standard genetic testing was inconclusive. Researchers analyzed DNA and RNA from blood samples of 56 participants, including patients and their family members. The goal w…
Matched conditions: LYSOSOMAL STORAGE DISEASES
Sponsor: Duke University • Aim: Knowledge-focused
Last updated Mar 25, 2026 14:08 UTC