Long-Term Improvements in Patient-Reported Outcomes in Patients with Pyruvate Kinase Deficiency Treated with Mitapivat

Kevin H. M. Kuo,Rachael F. Grace,Eduard J. van Beers,Wilma Barcellini,Andreas Glenthoj,Susanne Holzhauer,Vanessa Beynon,Susan Morris,Junlong Li,Erin Zagadailov,Parija Patel,Hanny Al-Samkari

American journal of hematology（2024）

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摘要

Clinically meaningful benefits in the signs, symptoms, and impacts of #PKDeficiency as assessed by disease-specific patient-reported outcome measures were observed in mitapivat-treated adult patients in two phase 3 clinical trials.

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Erythropoiesis-Stimulating Agents

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