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Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency
by
Kuo, Kevin H.M
, van Beers, Eduard J
, Barbier, Ann J
, Hua, Lei
, Layton, D. Mark
, Yaish, Hassan
, Jouvin, Marie-Hélène
, Morton, D. Holmes
, Bodie, Susan
, Glader, Bertil
, Rose, Christian
, Hawkins, Peter
, Sheth, Sujit
, Ravindranath, Yaddanapudi
, Grace, Rachael F
, Galactéros, Frédéric
, Kwiatkowski, Janet L
, Bowden, Chris
, Silver, Bruce
, Kung, Charles
, Barcellini, Wilma
in
Administration, Oral
/ Adolescent
/ Adult
/ Allosteric properties
/ Anemia
/ Anemia, Hemolytic, Congenital Nonspherocytic - blood
/ Anemia, Hemolytic, Congenital Nonspherocytic - drug therapy
/ Anemia, Hemolytic, Congenital Nonspherocytic - genetics
/ Catechols
/ Drug Administration Schedule
/ Female
/ Follow-Up Studies
/ Glucose
/ Headache
/ Headache - chemically induced
/ Hemoglobin
/ Hemoglobins - metabolism
/ Hemolytic anemia
/ Humans
/ Investigations
/ Kinases
/ Male
/ Mutation
/ Pharyngitis
/ Piperazines - administration & dosage
/ Piperazines - adverse effects
/ Pyruvate kinase
/ Pyruvate Kinase - blood
/ Pyruvate Kinase - deficiency
/ Pyruvate Kinase - genetics
/ Pyruvate Metabolism, Inborn Errors - blood
/ Pyruvate Metabolism, Inborn Errors - drug therapy
/ Pyruvate Metabolism, Inborn Errors - genetics
/ Pyruvic acid
/ Quinolines - administration & dosage
/ Quinolines - adverse effects
/ Sleep disorders
/ Sleep Initiation and Maintenance Disorders - chemically induced
/ Tyrphostins
/ Young Adult
2019
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Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency
by
Kuo, Kevin H.M
, van Beers, Eduard J
, Barbier, Ann J
, Hua, Lei
, Layton, D. Mark
, Yaish, Hassan
, Jouvin, Marie-Hélène
, Morton, D. Holmes
, Bodie, Susan
, Glader, Bertil
, Rose, Christian
, Hawkins, Peter
, Sheth, Sujit
, Ravindranath, Yaddanapudi
, Grace, Rachael F
, Galactéros, Frédéric
, Kwiatkowski, Janet L
, Bowden, Chris
, Silver, Bruce
, Kung, Charles
, Barcellini, Wilma
in
Administration, Oral
/ Adolescent
/ Adult
/ Allosteric properties
/ Anemia
/ Anemia, Hemolytic, Congenital Nonspherocytic - blood
/ Anemia, Hemolytic, Congenital Nonspherocytic - drug therapy
/ Anemia, Hemolytic, Congenital Nonspherocytic - genetics
/ Catechols
/ Drug Administration Schedule
/ Female
/ Follow-Up Studies
/ Glucose
/ Headache
/ Headache - chemically induced
/ Hemoglobin
/ Hemoglobins - metabolism
/ Hemolytic anemia
/ Humans
/ Investigations
/ Kinases
/ Male
/ Mutation
/ Pharyngitis
/ Piperazines - administration & dosage
/ Piperazines - adverse effects
/ Pyruvate kinase
/ Pyruvate Kinase - blood
/ Pyruvate Kinase - deficiency
/ Pyruvate Kinase - genetics
/ Pyruvate Metabolism, Inborn Errors - blood
/ Pyruvate Metabolism, Inborn Errors - drug therapy
/ Pyruvate Metabolism, Inborn Errors - genetics
/ Pyruvic acid
/ Quinolines - administration & dosage
/ Quinolines - adverse effects
/ Sleep disorders
/ Sleep Initiation and Maintenance Disorders - chemically induced
/ Tyrphostins
/ Young Adult
2019
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Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency
by
Kuo, Kevin H.M
, van Beers, Eduard J
, Barbier, Ann J
, Hua, Lei
, Layton, D. Mark
, Yaish, Hassan
, Jouvin, Marie-Hélène
, Morton, D. Holmes
, Bodie, Susan
, Glader, Bertil
, Rose, Christian
, Hawkins, Peter
, Sheth, Sujit
, Ravindranath, Yaddanapudi
, Grace, Rachael F
, Galactéros, Frédéric
, Kwiatkowski, Janet L
, Bowden, Chris
, Silver, Bruce
, Kung, Charles
, Barcellini, Wilma
in
Administration, Oral
/ Adolescent
/ Adult
/ Allosteric properties
/ Anemia
/ Anemia, Hemolytic, Congenital Nonspherocytic - blood
/ Anemia, Hemolytic, Congenital Nonspherocytic - drug therapy
/ Anemia, Hemolytic, Congenital Nonspherocytic - genetics
/ Catechols
/ Drug Administration Schedule
/ Female
/ Follow-Up Studies
/ Glucose
/ Headache
/ Headache - chemically induced
/ Hemoglobin
/ Hemoglobins - metabolism
/ Hemolytic anemia
/ Humans
/ Investigations
/ Kinases
/ Male
/ Mutation
/ Pharyngitis
/ Piperazines - administration & dosage
/ Piperazines - adverse effects
/ Pyruvate kinase
/ Pyruvate Kinase - blood
/ Pyruvate Kinase - deficiency
/ Pyruvate Kinase - genetics
/ Pyruvate Metabolism, Inborn Errors - blood
/ Pyruvate Metabolism, Inborn Errors - drug therapy
/ Pyruvate Metabolism, Inborn Errors - genetics
/ Pyruvic acid
/ Quinolines - administration & dosage
/ Quinolines - adverse effects
/ Sleep disorders
/ Sleep Initiation and Maintenance Disorders - chemically induced
/ Tyrphostins
/ Young Adult
2019
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Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency
Journal Article
Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency
2019
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Overview
In this study, approximately half the patients with red-cell pyruvate kinase deficiency who were treated with mitapivat had an improvement in their hemoglobin level and decreased hemolysis that was sustained for nearly 3 years. Patients who had missense mutations that allowed for synthesis of a hypofunctioning enzyme were most likely to have a response.
Publisher
Massachusetts Medical Society
Subject
/ Adult
/ Anemia
/ Anemia, Hemolytic, Congenital Nonspherocytic - blood
/ Anemia, Hemolytic, Congenital Nonspherocytic - drug therapy
/ Anemia, Hemolytic, Congenital Nonspherocytic - genetics
/ Drug Administration Schedule
/ Female
/ Glucose
/ Headache
/ Headache - chemically induced
/ Humans
/ Kinases
/ Male
/ Mutation
/ Piperazines - administration & dosage
/ Piperazines - adverse effects
/ Pyruvate Kinase - deficiency
/ Pyruvate Metabolism, Inborn Errors - blood
/ Pyruvate Metabolism, Inborn Errors - drug therapy
/ Pyruvate Metabolism, Inborn Errors - genetics
/ Quinolines - administration & dosage
/ Quinolines - adverse effects
/ Sleep Initiation and Maintenance Disorders - chemically induced
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