DRIVE PK: A Phase 2 Trial of AG-348 in Patients with Pyruvate Kinase Deficiency

被引：0
|
作者：
Barbier, Ann [1 ]
Silver, Bruce [2 ]
Merica, Elizabeth [1 ]
Cohen, Marvin [3 ]
Kung, Charles [1 ]
Yang, Hua [1 ]
Grace, Rachael F. [4 ]
Glader, Bertil [5 ]
Agresta, Sam [1 ]
机构：
[1] Agios Pharmaceut, Cambridge, MA USA
[2] Bruce A Silver Clin Sci & Dev, Dunkirk, MD USA
[3] MBC Pharma Solut, Newtown, PA USA
[4] Harvard Univ, Sch Med, Dana Farber Boston Childrens Canc & Blood Disorde, Boston, MA USA
[5] Stanford Univ, Lucile Packard Childrens Hosp, Palo Alto, CA 94304 USA
来源：
BLOOD | 2015年 / 126卷 / 23期
关键词：
D O I：
暂无
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
引用
收藏
页数：3
相关论文
共 50 条
  • [41] Pyruvate Kinase (PK) Protein and Enzyme Levels in the Diagnosis and Clinical Phenotype of PK Deficiency
    Addonizio, Kathryn
    Al-Samkari, Hanny
    Glader, Bertil
    Morton, D. Holmes
    Chonat, Satheesh
    Thompson, Alexis A.
    Kuo, Kevin H. M.
    Ravindranath, Yaddanapudi
    Wang, Heng
    Rothman, Jennifer A.
    Kwiatkowski, Janet L.
    Kung, Charles
    Kosinski, Penelope A.
    Al-Sayegh, Hasan
    London, Wendy B.
    Grace, Rachael F.
    BLOOD, 2019, 134
  • [42] Phase 1 Single- and Multiple-Ascending-Dose Randomized Studies of the Safety, Pharmacokinetics, and Pharmacodynamics of AG-348, a First-in-Class Allosteric Activator of Pyruvate Kinase R, in Healthy Volunteers
    Yang, Hua
    Merica, Elizabeth
    Chen, Yue
    Cohen, Marvin
    Goldwater, Ronald
    Kosinski, Penelope A.
    Kung, Charles
    Yuan, Zheng
    Silverman, Lee
    Goldwasser, Meredith
    Silver, Bruce A.
    Agresta, Sam
    Barbier, Ann J.
    CLINICAL PHARMACOLOGY IN DRUG DEVELOPMENT, 2019, 8 (02): : 246 - 259
  • [43] AG-348 (mitapivat), an allosteric activator of red blood cell pyruvate kinase, increases enzymatic activity, protein stability, and adenosine triphosphate levels over a broad range of PKLR genotypes
    Rab, Minke A. E.
    van Oirschot, Brigitte A.
    Kosinski, Penelope A.
    Hixon, Jeffrey
    Johnson, Kendall
    Chubukov, Victor
    Dang, Lenny
    Pasterkamp, Gerard
    van Straaten, Stephanie
    van Solinge, Wouter W.
    van Beers, Eduard J.
    Kung, Charles
    van Wijk, Richard
    HAEMATOLOGICA, 2021, 106 (01) : 238 - 249
  • [44] STUDIES ON PYRUVATE-KINASE (PK) DEFICIENCY .2. ELECTROPHORETIC, KINETIC, AND IMMUNOLOGICAL STUDIES ON PYRUVATE-KINASE OF ERYTHROCYTES AND OTHER TISSUES
    IMAMURA, K
    TANAKA, T
    NISHINA, T
    NAKASHIMA, K
    MIWA, S
    JOURNAL OF BIOCHEMISTRY, 1973, 74 (06): : 1165 - 1175
  • [45] The pyruvate kinase (PK) to hexokinase enzyme activity ratio and erythrocyte PK protein level in the diagnosis and phenotype of PK deficiency
    Al-Samkari, Hanny
    Addonizio, Kathryn
    Glader, Bertil
    Morton, D. Holmes
    Chonat, Satheesh
    Thompson, Alexis A.
    Kuo, Kevin H. M.
    Ravindranath, Yaddanapudi
    Wang, Heng
    Rothman, Jennifer A.
    Kwiatkowski, Janet L.
    Kung, Charles
    Kosinski, Penelope A.
    Al-Sayegh, Hasan
    London, Wendy B.
    Grace, Rachael F.
    BRITISH JOURNAL OF HAEMATOLOGY, 2021, 192 (06) : 1092 - 1096
  • [46] ERYTHROCYTE PYRUVATE-KINASE DEFICIENCY - CHARACTERIZATION OF A NEW VARIANT (PK AARAU)
    LAKOMEK, M
    WINKLER, H
    SCHARNETZKY, M
    TILLMANN, W
    LAIER, G
    MARTI, HR
    SCHROTER, W
    BLUT, 1984, 48 (03): : 123 - 129
  • [47] FOUR NOVEL PKLR GENE MUTATIONS IN ASSOCIATION WITH PYRUVATE KINASE (PK) DEFICIENCY
    Manco, L.
    Relvas, L.
    Rebelo, U.
    Murga, M. J.
    Santos, A.
    Yanez, L.
    Bento, C.
    Ribeiro, M. L.
    HAEMATOLOGICA-THE HEMATOLOGY JOURNAL, 2010, 95 : 430 - 430
  • [48] PK Aarau:: first homozygous nonsense mutation causing pyruvate kinase deficiency
    Sedano, IB
    Röthlisberger, B
    Délèze, G
    Ottiger, C
    Panchard, MA
    Spahr, A
    Hergersberg, M
    Bürgi, W
    Huber, A
    BRITISH JOURNAL OF HAEMATOLOGY, 2004, 127 (03) : 364 - 366
  • [49] CHANGE OF PYRUVATE-KINASE (PK) ISOZYMES IN CLASSICAL TYPE PK DEFICIENCY AND OTHER PK DEFICIENCY CASES DURING RED-CELL MATURATION
    TAKEGAWA, S
    MIWA, S
    AMERICAN JOURNAL OF HEMATOLOGY, 1984, 16 (01) : 53 - 58
  • [50] Phenotypic Corrections of Ex Vivo Erythrocytes Derived from Chronic Hemolytic Anemia Patients Due to Kruppel-like Factor 1 (KLF-1) Mutations By AG-348: A Novel Synthetic Pyruvate Kinase Activator
    Suksangpleng, Thidarat
    Tachavanich, Kalaya
    Riolueang, Suchada
    Ekwattanakit, Supachai
    Viprakasit, Vip
    BLOOD, 2019, 134
12345