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Journal Articles

Combined Red Cell and Plasma Exchange Is Associated with Reduced Mortality of Fat Embolism Syndrome in Sickle Cell Disease

Aushna Rasool, Sinju Thomas, Asad Luqmani, Mamta Sohal, Steven Okoli, Simona Deplano, Christine Ademokun, Catriona Mactier, Nichola Cooper, D. Mark Layton

Blood (2024) 144 (Supplement 1): 2497.

https://doi.org/10.1182/blood-2024-203179

Published: 2024

Journal Articles

Long-Term Hemoglobin Response and Reduction in Transfusion Burden Are Maintained in Patients with Pyruvate Kinase Deficiency Treated with Mitapivat

Blood (2022) 140 (Supplement 1): 5313–5315.

https://doi.org/10.1182/blood-2022-169125

Published: 2022

Journal Articles

Mitapivat Improves Markers of Erythropoietic Activity in Long-Term Study of Adults with Alpha- or Beta-Non-Transfusion-Dependent Thalassemia

Kevin H.M. Kuo, D. Mark Layton, Ashutosh Lal, Hanny Al-Samkari, Penelope A. Kosinski, Bo Tong, Jeremie H. Estepp, Katrin Uhlig, Elliott P. Vichinsky

Blood (2022) 140 (Supplement 1): 2479–2480.

https://doi.org/10.1182/blood-2022-163493

Published: 2022

Journal Articles

The Launch of Two Sub-Studies of the Peak Registry, a Global, Longitudinal Study of Pyruvate Kinase Deficiency

Carl Lander, Rachael F. Grace, Eduard J. van Beers, Joan-Lluis Vives Corrons, Bert Glader, Kevin H.M. Kuo, Andreas Glenthøj, Dagmar Pospíšilová, D. Mark Layton, Audra Boscoe, Stephanie Smith, Bryan McGee, Paola Bianchi

Blood (2022) 140 (Supplement 1): 11041–11042.

https://doi.org/10.1182/blood-2022-163079

Published: 2022

Journal Articles

Characterizing Iron Overload By Age in Patients Diagnosed with Pyruvate Kinase Deficiency - a Descriptive Analysis from the Peak Registry

Paola Bianchi, Rachael F. Grace, Joan-Lluis Vives Corrons, Bert Glader, Andreas Glenthøj, Hitoshi Kanno, Kevin H.M. Kuo, Carl Lander, D. Mark Layton, Dagmar Pospíŝilová, Vip Viprakasit, Jean Williams, Yan Yan, Bryan McGee, Eduard J. Van Beers

Blood (2021) 138 (Supplement 1): 3074.

https://doi.org/10.1182/blood-2021-147294

Published: 2021

Journal Articles

Long-Term Efficacy and Safety of the Oral Pyruvate Kinase Activator Mitapivat in Adults with Non-Transfusion-Dependent Alpha- or Beta-Thalassemia

Kevin H.M. Kuo, D. Mark Layton, Ashutosh Lal, Hanny Al-Samkari, Joy Bhatia, Penelope A. Kosinski, Bo Tong, Megan Lynch, Katrin Uhlig, Elliott Vichinsky

Blood (2021) 138 (Supplement 1): 576.

https://doi.org/10.1182/blood-2021-150386

Published: 2021

Journal Articles

Durability of Hemoglobin Response and Reduction in Transfusion Burden Is Maintained over Time in Patients with Pyruvate Kinase Deficiency Treated with Mitapivat in a Long-Term Extension Study

Blood (2021) 138 (Supplement 1): 848.

https://doi.org/10.1182/blood-2021-147711

Published: 2021

Journal Articles

Bone Mineral Density Remains Stable in Pyruvate Kinase Deficiency Patients Receiving Long-Term Treatment with Mitapivat

Hanny Al-Samkari, Rachael F. Grace, Andreas Glenthoej, Oliver Andres, Wilma Barcellini, Frédéric Galactéros, Kevin H.M. Kuo, D. Mark Layton, Marta Morado, Vip Viprakasit, Yan Dong, Feng Tai, Liz Grekas, Karishma Khoja, Sarah Gheuens, Bryan McGee, John B. Porter, Eduard J. Van Beers

Blood (2021) 138 (Supplement 1): 924.

https://doi.org/10.1182/blood-2021-147156

Published: 2021

Journal Articles

Baseline Characteristics of Patients in Peak: A Global, Longitudinal Registry of Patients with Pyruvate Kinase Deficiency

Rachael F. Grace, MD, Audra Boscoe, PhD, Chris Bowden, MD, Bertil Glader, MD PhD, Hitoshi Kanno, MDPhD, D. Mark Layton, MB BS, Eduard van Beers, MD PhD, Joan-Lluis Vives Corrons, MD PhD, Yan Yan, MS, Paola Bianchi, BSc, PhD

Blood (2020) 136 (Supplement 1): 39–40.

https://doi.org/10.1182/blood-2020-136634

Published: 2020

Journal Articles

Early-Onset Osteopenia and Osteoporosis in Patients with Pyruvate Kinase Deficiency

Hanny Al-Samkari, MD, Rachael F. Grace, MD, Andreas Glenthoej, MD, Oliver Andres, MD, Wilma Barcellini, Frédéric Galactéros, MDPhD, Kevin H.M. Kuo, MDMSc,FRCPC, D. Mark Layton, MB BS, Marta Morado, PhD, Vip Viprakasit, Yan Dong, PhD, Feng Tai, PhD, Peter Hawkins, PhD, Sarah Gheuens, MD PhD, Chris Bowden, MD, John B. Porter, MA, MD, FRCP, FRCPath, Eduard van Beers, MD PhD

Blood (2020) 136 (Supplement 1): 30–32.

https://doi.org/10.1182/blood-2020-136598

Published: 2020

Journal Articles

An Ongoing Global, Longitudinal, Observational Study of Patients with Pyruvate Kinase Deficiency: The PEAK Registry

Rachael F. Grace, MD, Paola Bianchi, BSc, PhD, Bertil Glader, MD PhD, Andreas Glenthøj, MD PhD, Bryan Jones, PhD, Hitoshi Kanno, MD PhD, Kevin H. M. Kuo, MDMSc,FRCPC, D. Mark Layton, MB ChB, Eduard J. van Beers, MD PhD, Joan-Lluis Vives Corrons, MD PhD, Tu Xu, MS, PhD

Blood (2019) 134 (Supplement_1): 2223.

https://doi.org/10.1182/blood-2019-123059

Published: 2019

Journal Articles

Mitapivat (AG-348), an Oral PK-R Activator, in Adults with Non-Transfusion Dependent Thalassemia: A Phase 2, Open-Label, Multicenter Study in Progress

Kevin H. M. Kuo, MDMSc,FRCPC, D. Mark Layton, MB BS, Katrin Uhlig, MD MS, Megan Lynch, MSN, NP, Li Liu, PhD, Elliott P. Vichinsky, MD

Blood (2019) 134 (Supplement_1): 2249.

https://doi.org/10.1182/blood-2019-123113

Published: 2019

Journal Articles

Long-Term Safety and Efficacy of Mitapivat (AG-348), a Pyruvate Kinase Activator, in Patients with Pyruvate Kinase Deficiency: The DRIVE PK Study

Rachael F. Grace, MD, D. Mark Layton, MB BS, FRCP, FRCPCH, Frédéric Galactéros, MD PhD, Wilma Barcellini, MD, Eduard J. van Beers, MD PhD, Hassan M. Yaish, MD, Yaddanapudi Ravindranath, MBBS, Kevin H. M. Kuo, MDMSc,FRCPC, Sujit Sheth, MD, Janet L. Kwiatkowski, MD MSCE, Lei Hua, PhD, Peter F. Hawkins, PhD, Chris Mix, MD, Bertil Glader, MD PhD

Blood (2019) 134 (Supplement_1): 3512.

https://doi.org/10.1182/blood-2019-123406

Published: 2019

Journal Articles

A phase 1/2 ascending dose study and open-label extension study of voxelotor in patients with sickle cell disease

Jo Howard, Claire Jane Hemmaway, Paul Telfer, D. Mark Layton, John Porter, Moji Awogbade, Timothy Mant, Daniel D. Gretler, Kobina Dufu, Athiwat Hutchaleelaha, Mira Patel, Vincent Siu, Sandra Dixon, Noel Landsman, Margaret Tonda, Joshua Lehrer-Graiwer

Blood (2019) 133 (17): 1865–1875.

https://doi.org/10.1182/blood-2018-08-868893

Published: 2019

Journal Articles

Results Update from the DRIVE PK Study: Effects of AG-348, a Pyruvate Kinase Activator, in Patients with Pyruvate Kinase Deficiency

Blood (2017) 130 (Supplement 1): 2194.

https://doi.org/10.1182/blood.V130.Suppl_1.2194.2194

Published: 2017

Journal Articles

Effects of AG-348, a Pyruvate Kinase Activator, on Anemia and Hemolysis in Patients with Pyruvate Kinase Deficiency: Data from the DRIVE PK Study

Blood (2016) 128 (22): 402.

https://doi.org/10.1182/blood.V128.22.402.402

Published: 2016

Journal Articles

Cell-type–specific transcriptional regulation of PIGM underpins the divergent hematologic phenotype in inherited GPl deficiency

Joana R. Costa, Valentina S. Caputo, Kalliopi Makarona, D. Mark Layton, Irene A. G. Roberts, Antonio M. Almeida, Anastasios Karadimitris

Blood (2014) 124 (20): 3151–3154.

https://doi.org/10.1182/blood-2014-09-598813

Published: 2014

Includes: Supplemental data

Journal Articles

Transcriptional and epigenetic basis for restoration of G6PD enzymatic activity in human G6PD-deficient cells

Kalliopi Makarona, Valentina S. Caputo, Joana R. Costa, Binbin Liu, David O’Connor, Deena Iskander, David Roper, Lynn Robertson, Neha Bhatnagar, Evangelos Terpos, Elisabeth Georgiou, Maria Papaioannou, D. Mark Layton, Lucio Luzzatto, Irene Roberts, Anastasios Karadimitris

Blood (2014) 124 (1): 134–141.

https://doi.org/10.1182/blood-2014-02-553792

Published: 2014

Includes: Supplemental data

Journal Articles

Protein 4.2 is critical to CD47-membrane skeleton attachment in human red cells

Kris Noel Dahl, Ranganath Parthasarathy, Connie M. Westhoff, D. Mark Layton, Dennis E. Discher

Blood (2004) 103 (3): 1131–1136.

https://doi.org/10.1182/blood-2003-04-1331

Published: 2004

Journal Articles

Absence of CD47 in protein 4.2–deficient hereditary spherocytosis in man: an interaction between the Rh complex and the band 3 complex: Presented at the 43rd Annual Meeting of the American Society of Hematology, Orlando, FL, December 7-11, 2001, and abstract published in Blood. 2001;98:(suppl1):10a.

Lesley J. Bruce, Sandip Ghosh, May Jean King, D. Mark Layton, William J. Mawby, Gordon W. Stewart, Per-Arne Oldenborg, Jean Delaunay, Michael J. A. Tanner

Blood (2002) 100 (5): 1878–1885.

https://doi.org/10.1182/blood-2002-03-0706

Published: 2002

NARROW

Combined Red Cell and Plasma Exchange Is Associated with Reduced Mortality of Fat Embolism Syndrome in Sickle Cell Disease

Long-Term Hemoglobin Response and Reduction in Transfusion Burden Are Maintained in Patients with Pyruvate Kinase Deficiency Treated with Mitapivat

Mitapivat Improves Markers of Erythropoietic Activity in Long-Term Study of Adults with Alpha- or Beta-Non-Transfusion-Dependent Thalassemia

The Launch of Two Sub-Studies of the Peak Registry, a Global, Longitudinal Study of Pyruvate Kinase Deficiency

Characterizing Iron Overload By Age in Patients Diagnosed with Pyruvate Kinase Deficiency - a Descriptive Analysis from the Peak Registry

Long-Term Efficacy and Safety of the Oral Pyruvate Kinase Activator Mitapivat in Adults with Non-Transfusion-Dependent Alpha- or Beta-Thalassemia

Durability of Hemoglobin Response and Reduction in Transfusion Burden Is Maintained over Time in Patients with Pyruvate Kinase Deficiency Treated with Mitapivat in a Long-Term Extension Study

Bone Mineral Density Remains Stable in Pyruvate Kinase Deficiency Patients Receiving Long-Term Treatment with Mitapivat

Baseline Characteristics of Patients in Peak: A Global, Longitudinal Registry of Patients with Pyruvate Kinase Deficiency

Early-Onset Osteopenia and Osteoporosis in Patients with Pyruvate Kinase Deficiency

An Ongoing Global, Longitudinal, Observational Study of Patients with Pyruvate Kinase Deficiency: The PEAK Registry

Mitapivat (AG-348), an Oral PK-R Activator, in Adults with Non-Transfusion Dependent Thalassemia: A Phase 2, Open-Label, Multicenter Study in Progress

Long-Term Safety and Efficacy of Mitapivat (AG-348), a Pyruvate Kinase Activator, in Patients with Pyruvate Kinase Deficiency: The DRIVE PK Study

A phase 1/2 ascending dose study and open-label extension study of voxelotor in patients with sickle cell disease

Results Update from the DRIVE PK Study: Effects of AG-348, a Pyruvate Kinase Activator, in Patients with Pyruvate Kinase Deficiency

Effects of AG-348, a Pyruvate Kinase Activator, on Anemia and Hemolysis in Patients with Pyruvate Kinase Deficiency: Data from the DRIVE PK Study

Cell-type–specific transcriptional regulation of PIGM underpins the divergent hematologic phenotype in inherited GPl deficiency

Transcriptional and epigenetic basis for restoration of G6PD enzymatic activity in human G6PD-deficient cells

Protein 4.2 is critical to CD47-membrane skeleton attachment in human red cells

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American Society of Hematology

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ASH Publications

American Society of Hematology

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