The New England journal of medicine, 2019-09, Vol.381 (10), p.933-944
- Grace, Rachael F
- Rose, Christian
- Layton, D. Mark
- Galactéros, Frédéric
- Barcellini, Wilma
- Morton, D. Holmes
- van Beers, Eduard J
- Yaish, Hassan
- Ravindranath, Yaddanapudi
- Kuo, Kevin H.M
- Sheth, Sujit
- Kwiatkowski, Janet L
- Barbier, Ann J
- Bodie, Susan
- Silver, Bruce
- Hua, Lei
- Kung, Charles
- Hawkins, Peter
- Jouvin, Marie-Hélène
- Bowden, Chris
- Glader, Bertil
2019
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- Autor(en) / Beteiligte
- Grace, Rachael F
- Rose, Christian
- Layton, D. Mark
- Galactéros, Frédéric
- Barcellini, Wilma
- Morton, D. Holmes
- van Beers, Eduard J
- Yaish, Hassan
- Ravindranath, Yaddanapudi
- Kuo, Kevin H.M
- Sheth, Sujit
- Kwiatkowski, Janet L
- Barbier, Ann J
- Bodie, Susan
- Silver, Bruce
- Hua, Lei
- Kung, Charles
- Hawkins, Peter
- Jouvin, Marie-Hélène
- Bowden, Chris
- Glader, Bertil
- Titel
- Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency
- Ist Teil von
- The New England journal of medicine, 2019-09, Vol.381 (10), p.933-944
- Ort / Verlag
- United States: Massachusetts Medical Society
- Erscheinungsjahr
- 2019
- Quelle
- MEDLINE
- Beschreibungen/Notizen
- 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.
- Sprache
- Englisch
- Identifikatoren
- ISSN: 0028-4793eISSN: 1533-4406DOI: 10.1056/NEJMoa1902678Titel-ID: cdi_proquest_miscellaneous_2285104036
- Format
- –
- Schlagworte
- 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