BETA-THALASSEMIA MAJOR
Clinical trials for BETA-THALASSEMIA MAJOR explained in plain language.
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One-Shot gene therapy aims to cure devastating blood disorder
⭐️ CURE ⭐️ CompletedThis early-stage study tested a new one-time gene therapy called CS-101 in 9 people with beta-thalassemia major, a severe inherited blood disease. Doctors collected each patient's own blood stem cells, used gene editing to fix the underlying genetic cause, and then gave them back…
Matched conditions: BETA-THALASSEMIA MAJOR
Phase: PHASE1 • Sponsor: CorrectSequence Therapeutics Co., Ltd • Aim: ⭐️ CURE ⭐️
Last updated Mar 30, 2026 14:32 UTC
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New hope for spotting silent liver damage in kids with blood disorder
Diagnosis CompletedThis study aimed to find better, non-invasive ways to check for liver scarring (fibrosis) in children with beta-thalassemia, a blood disorder that often causes iron overload. Researchers compared simple blood tests and a special scan against each other to see which was most accur…
Matched conditions: BETA-THALASSEMIA MAJOR
Sponsor: Tanta University • Aim: Diagnosis
Last updated Mar 06, 2026 15:37 UTC