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 thalassemia: first human test shows promise

Disease control Ongoing

This early-stage study tests a gene therapy for adults with β-thalassemia major, a severe blood disorder requiring frequent transfusions. Doctors take the patient's own stem cells, fix the faulty gene, and return the corrected cells via an IV. A low dose of busulfan prepares the …

Matched conditions: CONFIRMED DIAGNOSIS OF SS-THALASSEMIA MAJOR

Phase 1 • Sponsor: San Rocco Therapeutics • Aim: Disease control

Last updated Jun 27, 2026 12:23 UTC

Confirmed diagnosis of SS-thalassemia major

Get alerted when new Confirmed diagnosis of SS-thalassemia major trials appear

Gene fix for thalassemia: first human test shows promise