BETA THALASSEMIA MAJOR
Clinical trials for BETA THALASSEMIA MAJOR explained in plain language.
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Gene therapy breakthrough offers hope for blood disorder patients
Disease control CompletedThis early-stage study tested a single dose of CS-101 gene therapy in 9 people with beta-thalassemia major, a severe blood disorder requiring lifelong transfusions. The goal was to see if the treatment is safe and can help the body produce healthy hemoglobin. While promising, pat…
Matched conditions: BETA THALASSEMIA MAJOR
Phase: PHASE1 • Sponsor: CorrectSequence Therapeutics Co., Ltd • Aim: Disease control
Last updated May 15, 2026 11:56 UTC
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New test could improve iron monitoring in thalassemia
Knowledge-focused CompletedThis study looked at a new way to measure iron buildup inside red blood cells of people with beta thalassemia major, a serious blood disorder. The goal was to see if this method could be simpler and more direct than the usual blood test for ferritin, which has some drawbacks. Res…
Matched conditions: BETA THALASSEMIA MAJOR
Sponsor: Ankara City Hospital Bilkent • Aim: Knowledge-focused
Last updated May 17, 2026 04:44 UTC
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New study seeks best way to spot liver damage in thalassemia kids
Knowledge-focused CompletedThis study looked at 100 children with beta-thalassemia major and iron overload to see which tests best detect liver scarring. Researchers compared blood markers (ferritin, hepcidin) and non-invasive scores (APRI, FIB-4) against a special ultrasound (FibroScan). The goal was to f…
Matched conditions: BETA THALASSEMIA MAJOR
Sponsor: Tanta University • Aim: Knowledge-focused
Last updated May 17, 2026 04:43 UTC