A randomized phase I/II trial of HQK-1001, an oral fetal globin gene inducer, in β-thalassaemia intermedia and HbE/β-thalassaemia

• Main Authors: Suthat Fucharoen, Adlette Inati, Noppadol Siritanaratku, Swee L. Thein, William C. Wargin, Suzanne Koussa, Ali Taher, Nattawara Chaneim, Michael Boosalis, Ronald Berenson, Susan P. Perrine
• Other Authors: Mahidol University
• Format: Article
• Published: 2018
• Subjects: Medicine
• Online Access: https://repository.li.mahidol.ac.th/handle/123456789/32376

β-thalassaemia intermedia (BTI) syndromes cause haemolytic anaemia, ineffective erythropoiesis, and widespread complications. Higher fetal globin expression within genotypes reduces globin imbalance and ameliorates anaemia. Sodium 2,2 dimethylbutyrate (HQK-1001), an orally bioavailable short-chain f...