Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency

Author:

Grace Rachael F.1,Rose Christian1,Layton D. Mark1,Galactéros Frédéric1,Barcellini Wilma1,Morton D. Holmes1,van Beers Eduard J.1,Yaish Hassan1,Ravindranath Yaddanapudi1,Kuo Kevin H.M.1,Sheth Sujit1,Kwiatkowski Janet L.1,Barbier Ann J.1,Bodie Susan1,Silver Bruce1,Hua Lei1,Kung Charles1,Hawkins Peter1,Jouvin Marie-Hélène1,Bowden Chris1,Glader Bertil1

Affiliation:

1. From the Dana–Farber/Boston Children’s Cancer and Blood Disorders Center and Harvard Medical School, Boston (R.F.G.), and Agios Pharmaceuticals, Cambridge (A.J.B., S.B., L.H., C.K., P.H., M.-H.J., C.B.) — all in Massachusetts; Hôpital Saint Vincent de Paul, Lille (C.R.), and Unité des Maladies Génétiques du Globule Rouge, Centre Hospitalier Universitaire Henri Mondor, Créteil (F.G.) — both in France; Hammersmith Hospital, Imperial College Healthcare NHS Trust, London (D.M.L.); Fondazione IRCCS Ca’ Granda...

Funder

Agios Pharmaceuticals, Inc.

Publisher

Massachusetts Medical Society

Subject

General Medicine

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