BETA THALASSEMIA MAJOR
Clinical trials for BETA THALASSEMIA MAJOR explained in plain language.
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One-Time gene injection aims to free patients from lifelong blood transfusions
Disease control CompletedThis early-stage study tested a new, one-time gene therapy called CS-101 in 9 patients with a severe inherited blood disorder called beta-thalassemia major. The goal was to see if the treatment was safe and if it could help the patients' bodies make healthy red blood cells on the…
Matched conditions: BETA THALASSEMIA MAJOR
Phase: PHASE1 • Sponsor: CorrectSequence Therapeutics Co., Ltd • Aim: Disease control
Last updated Apr 04, 2026 06:19 UTC
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Searching for a better liver test for kids with blood disorder
Diagnosis CompletedThis study aimed to find the best non-invasive method to diagnose liver scarring (fibrosis) in children with beta-thalassemia major who have iron overload. Researchers compared simple blood tests and a special ultrasound scan (Fibroscan) in 100 children. The goal was to see if ce…
Matched conditions: BETA THALASSEMIA MAJOR
Sponsor: Tanta University • Aim: Diagnosis
Last updated Apr 04, 2026 06:22 UTC
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Scientists develop simpler test to track dangerous iron buildup
Knowledge-focused CompletedThis study tested a new laboratory method to measure iron that builds up inside the red blood cells of people with beta thalassemia major. The goal was to find a simpler, more direct way to monitor iron overload compared to current blood tests. Researchers analyzed blood samples …
Matched conditions: BETA THALASSEMIA MAJOR
Sponsor: Ankara City Hospital Bilkent • Aim: Knowledge-focused
Last updated Apr 03, 2026 14:41 UTC