LYSOSOMAL STORAGE DISEASES
Clinical trials for LYSOSOMAL STORAGE DISEASES explained in plain language.
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Experimental gene therapy aims to fix cystinosis at the source
Disease control CompletedThis early-phase trial tested a gene therapy for cystinosis, a rare genetic disorder that causes cystine crystals to build up in cells, damaging organs. Researchers took blood stem cells from 6 patients, added a working copy of the faulty gene, and infused the corrected cells bac…
Matched conditions: LYSOSOMAL STORAGE DISEASES
Phase: PHASE1, PHASE2 • Sponsor: University of California, San Diego • Aim: Disease control
Last updated Jun 27, 2026 08:12 UTC
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Scientists hunt for missing genetic clues in mystery diseases
Knowledge-focused CompletedThis study aimed to find new genetic changes that might cause inherited diseases, especially in people who already have a diagnosis but whose genetic tests were not clear. Researchers studied 56 patients and their family members to look for hidden variants in parts of the DNA tha…
Matched conditions: LYSOSOMAL STORAGE DISEASES
Sponsor: Duke University • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:11 UTC