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Journal Articles

Results from the Beacon Trial: A Phase 2, Randomized, Open-Label Study of Bitopertin in Erythropoietic Protoporphyria

Gayle Ross, Peter Stewart, George Mensing, Melanie Chin, Haley Howell, Heidi Mangus, Will Savage
Journal: Blood
Blood (2024) 144 (Supplement 1): 1093.
https://doi.org/10.1182/blood-2024-203387
Published: 2024
Journal Articles

Interim Analyses from the Beacon Trial: A Phase 2, Randomized, Open-Label Trial of Bitopertin in Erythropoietic Protoporphyria

Gayle Ross, Peter Stewart, George Mensing, Melanie Chin, Haley Howell, Heidi Mangus, William Savage
Journal: Blood
Blood (2023) 142 (Supplement 1): 923.
https://doi.org/10.1182/blood-2023-175011
Published: 2023
Journal Articles

Application of a Validated Method for Quantifying Circulating Protoporphyrin IX to the Beacon Trial of Bitopertin in Erythropoietic Protoporphyria

Heidi Mangus, George Mensing, William Savage, Melanie Chin, Yue Chen, Guowen Liu, Hua Yang
Journal: Blood
Blood (2023) 142 (Supplement 1): 5228.
https://doi.org/10.1182/blood-2023-178122
Published: 2023
Journal Articles

Pklr Variants Associated with Acute Pain in Sickle Cell Disease Influence ATP Concentrations in Red Blood Cells

Xunde Wang, Yi-Ping Fu, Quan Li, Emily B Dunkelberger, Mickias Tegegn, Penelope A. Kosinski, Heidi Mangus, John Hamre, Sam M. Mbulaiteye, Ludmila Prokunina-Olsson, Stephen J. Chanock, Nancy Asomaning, Neal Jeffries, William Eaton, Swee Lay Thein
Journal: Blood
Blood (2022) 140 (Supplement 1): 954–956.
https://doi.org/10.1182/blood-2022-167296
Published: 2022
Journal Articles

A phase 1 dose escalation study of the pyruvate kinase activator mitapivat (AG-348) in sickle cell disease

Clinical Trials & Observations
Julia Z. Xu, Anna Conrey, Ingrid Frey, Eveline Gwaabe, Laurel A. Menapace, Laxminath Tumburu, Maureen Lundt, Timothy Lequang, Quan Li, Kristen Glass, Emily B. Dunkelberger, Varsha Iyer, Heidi Mangus, Charles Kung, Lenny Dang, Penelope A. Kosinski, Peter Hawkins, Neal Jeffries, William A. Eaton, Swee Lay Thein
Journal: Blood
Blood (2022) 140 (19): 2053–2062.
https://doi.org/10.1182/blood.2022015403
Published: 2022
Includes: Supplemental data
Journal Articles

Mitapivat (AG-348) Demonstrates Safety, Tolerability, and Improvements in Anemia, Hemolysis, Oxygen Affinity, and Hemoglobin S Polymerization Kinetics in Adults with Sickle Cell Disease: A Phase 1 Dose Escalation Study

Julia Z. Xu, Anna Conrey, Ingrid Frey, Eveline Gwaabe, Laurel A Menapace, Laxminath Tumburu, Maureen Lundt, Quan Li, Kristen Glass, Varsha Iyer, Heidi Mangus, Charles Kung, Lenny Dang, Penelope A. Kosinski, Peter Hawkins, Neal Jeffries, William Eaton, Swee Lay Thein
Journal: Blood
Blood (2021) 138 (Supplement 1): 10.
https://doi.org/10.1182/blood-2021-145398
Published: 2021
Journal Articles

Decreased activity and stability of pyruvate kinase in sickle cell disease: a novel target for mitapivat therapy

Minke A. E. Rab, Jennifer Bos, Brigitte A. van Oirschot, Stephanie van Straaten, Penelope A. Kosinski, Victor Chubukov, Hyeryun Kim, Heidi Mangus, Roger E. G. Schutgens, Gerard Pasterkamp, Lenny Dang, Charles Kung, Eduard J. van Beers, Richard van Wijk
Journal: Blood
Blood (2021) 137 (21): 2997–3001.
https://doi.org/10.1182/blood.2020008635
Published: 2021
Includes: Supplemental data
Journal Articles

Phase 1 Multiple Ascending Dose Study of Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of Mitapivat (AG-348) in Subjects with Sickle Cell Disease

Julia Z. Xu, MDMS, Anna Conrey, NP, Ingrid Frey, RN, BSN, MS, James Nichols, RN, Laurel A Menapace, MD, Laxminath Tumburu, PhD, Timothy Lequang, MSc, Quan Li, PhD, Emily Dunkelberger, PhD, Eric Henry, PhD, Troy Cellmer, PhD, Varsha Iyer, Heidi Mangus, BS, Charles Kung, PhD, Lenny Dang, PhD, Penelope A. Kosinski, MS, Peter Hawkins, PhD, Neal Jeffries, PhD, William Eaton, MD, Swee Lay Thein, DSc, FRCP, MBBS, MRCP
Journal: Blood
Blood (2020) 136 (Supplement 1): 21–22.
https://doi.org/10.1182/blood-2020-137716
Published: 2020
Journal Articles

Genotype-Response Correlation in DRIVE PK, a Phase 2 Study of AG-348 in Patients with Pyruvate Kinase Deficiency

Charles Kung, PhD, Penelope A. Kosinski, MS, Heidi Mangus, BS, Lei Hua, PhD, Gary Connor, Michelle Mobilia, BS, Karen Sullivan, BA, Sasha Frye, BS, Marie-Hélène Jouvin, MD, Rachael F. Grace, MD, Bertil Glader, MD, Chris Bowden, MD
Journal: Blood
Blood (2018) 132 (Supplement 1): 3621.
https://doi.org/10.1182/blood-2018-99-111166
Published: 2018