Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency
Rachael F. Grace(Dana-Farber/Boston Children's Cancer and Blood Disorders Center), Bertil Glader(Stanford University), F. Galactéros(Université Paris-Est Créteil), Hassan M. Yaish(Primary Children's Hospital), Wilma Barcellini(Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico), Sujit Sheth(Columbia University Irving Medical Center), Bruce A. Silver(Johns Hopkins Hospital), Chris Bowden(Agios Pharmaceuticals (United States)), D. Holmes Morton(Clinic for Special Children), Yaddanapudi Ravindranath(The Barbara Ann Karmanos Cancer Institute), Kevin H.M. Kuo(University Health Network), Marie-Hélène Jouvin(Agios Pharmaceuticals (United States)), Peter Hawkins(Agios Pharmaceuticals (United States)), Janet L. Kwiatkowski(Children's Hospital of Philadelphia), Ann Barbier(Agios Pharmaceuticals (United States)), Christian Rosé(Klinik und Poliklinik für Hautkrankheiten), Lei Hua(Agios Pharmaceuticals (United States)), D. Mark Layton(Imperial College Healthcare NHS Trust), Eduard J. van Beers(Utrecht University), Charles Kung(Agios Pharmaceuticals (United States)), Susan Bodie(Agios Pharmaceuticals (United States))
Cited by 162
Related Papers
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
|Nature|2010|1.3k
Ribosomal Protein L5 and L11 Mutations Are Associated with Cleft Palate and Abnormal Thumbs in Diamond-Blackfan Anemia Patients
|The American Journal of Human Genetics|2008|417
Clinical heterogeneity and predictors of outcome in primary autoimmune hemolytic anemia: a GIMEMA study of 308 patients
|Blood|2014|373
Home Treatment of Mild to Moderate Bleeding Episodes Using Recombinant Factor VIIa (Novoseven) in Haemophiliacs with Inhibitors
|Thrombosis and Haemostasis|1998|355