Beta-thalassemia
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Sign up freeTop drugs studied for this disease
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EMA withdrawal · 2022-11-30EMA Withdrawn: Zynteglo (autologous CD34+ cell enriched population that contains haematopoietic stem cells transduced with lentiviral vector encoding the βA-T87Q-globin gene) — bluebird bio (Netherlands) B.V.
EMA withdrawn Zynteglo, a gene therapy for beta-thalassemia previously granted conditional approval.
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CLINICAL_TRIALS withdrawal · 2015-09-29Clinical Trial Update: Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-Thalassemia [Phase 1]
A Phase 1 safety and pharmacokinetic study of escalating doses of SP-420, an iron chelator, in patients with beta-thalassemia was terminated…