Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in adults with non-transfusion dependent α-thalassaemia or β-thalassaemia: an open-label, multicentre, phase 2 study
Kevin H.M. Kuo(University Health Network), Elliott Vichinsky(Unknown), Penelope A. Kosinski(Agios Pharmaceuticals (United States)), Hanny Al‐Samkari(Harvard University), Megan Suzanne Lynch(Agios Pharmaceuticals (United States)), Tong Bo(Agios Pharmaceuticals (United States)), Katrin Uhlig(Tufts Medical Center), Ashutosh Lal(Post Graduate Institute of Medical Education and Research), D. Mark Layton(Imperial College Healthcare NHS Trust), Joy Bhatia(Agios Pharmaceuticals (United States))
Cited by 87
Related Papers
Sickle cell disease
|Nature Reviews Disease Primers|2018|1.4k
A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent β-Thalassemia
|New England Journal of Medicine|2020|320
Clinical spectrum of pyruvate kinase deficiency: data from the Pyruvate Kinase Deficiency Natural History Study
|Blood|2018|165
Graft Use in Transvaginal Pelvic Organ Prolapse Repair
|Obstetrics and Gynecology|2008|163
Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency
|New England Journal of Medicine|2019|162