CONFIRMED DIAGNOSIS OF SS-THALASSEMIA MAJOR

Clinical trials for CONFIRMED DIAGNOSIS OF SS-THALASSEMIA MAJOR explained in plain language.

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Gene fix for blood disorder shows early promise in small trial

Disease control Ongoing

This early-stage study tests a gene therapy for people with beta-thalassemia major, a severe inherited blood disorder. Doctors take the patient's own stem cells, fix the faulty gene in the lab, and return the corrected cells through an IV. A low dose of busulfan is given beforeha…

Matched conditions: CONFIRMED DIAGNOSIS OF SS-THALASSEMIA MAJOR

Phase: PHASE1 • Sponsor: San Rocco Therapeutics • Aim: Disease control

Last updated May 17, 2026 10:07 UTC

CONFIRMED DIAGNOSIS OF SS-THALASSEMIA MAJOR

Get alerted when new CONFIRMED DIAGNOSIS OF SS-THALASSEMIA MAJOR trials appear

Gene fix for blood disorder shows early promise in small trial