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American Journal of Hematology|February 12, 2020
Genotype-phenotype correlation and molecular heterogeneity in pyruvate kinase deficiencyPaola Bianchi, Elisa Fermo, Kimberly Lezon-Geyda, et al.
The Lancet. Haematology|December 7, 2024
Safety and efficacy of mitapivat in sickle cell disease (RISE UP): results from the phase 2 portion of a global, double-blind, randomised, placebo-controlled trialModupe Idowu, Lucas Otieno, Bogdan Dumitriu, et al.
The Lancet. Haematology|February 8, 2024
Diagnosis and management of pyruvate kinase deficiency: international expert guidelinesHanny Al-Samkari, Nadine Shehata, Kelly Lang-Robertson, et al.
The New England Journal of Medicine|March 27, 2020
A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent β-ThalassemiaM Domenica Cappellini, Vip Viprakasit, Ali T Taher, et al.
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