Concepedia

Mitapivat in adult patients with pyruvate kinase deficiency receiving regular transfusions (ACTIVATE-T): a multicentre, open-label, single-arm, phase 3 trial

Andreas Glenthøj, Eduard J. van Beers, Hanny Al‐Samkari, Vip Viprakasit, Kevin H.M. Kuo, Frederic Galactéros, Satheesh Chonat, John B. Porter, Erin Zagadailov, Rengyi Xu,

The Lancet Haematology · 2022 · 48 citations · 10 references

DOI

Concepts

References

10

Clinical spectrum of pyruvate kinase deficiency: data from the Pyruvate Kinase Deficiency Natural History Study

Rachael F. Grace, Paola Bianchi, Eduard J. van Beers et al. · Blood · 2018 · 163 citations · Full text

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Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency

Rachael F. Grace, Christian Rosé, D. Mark Layton et al. · New England Journal of Medicine · 2019 · 159 citations · Full text

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Estimating the prevalence of pyruvate kinase deficiency from the gene frequency in the general white population

Ernest Beutler, Terri Gelbart · Blood · 2000 · 141 citations

, , +11

AG-348 enhances pyruvate kinase activity in red blood cells from patients with pyruvate kinase deficiency

Charles Kung, Jeff Hixon, Penelope A. Kosinski et al. · Blood · 2017 · 129 citations · Full text

, , +13

Luspatercept improves hemoglobin levels and blood transfusion requirements in a study of patients with β-thalassemia

Antonio Piga, Silverio Perrotta, Maria Rita Gamberini et al. · Blood · 2019 · 114 citations · Full text

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