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Author Notes:
Correspondence: rachael.grace@childrens.harvard.edu
RFG, EJvB, and SvS conceived the study, analyzed and interpreted the data, and wrote the manuscript.
DHM, WB, SWE, BG, HMY, SC, JLK, JAR, MS, EJN, and SS conceived the studies, interpreted the data and provided critical revisions to the manuscript.
JDM, NK, DP, CMK, KK, YDP, AAT, PEN, YR, WCW, MWW, HW, SH, VRB, MV, JK, MAM, MJR, HAB, and KA performed the research and contributed to the manuscript.
AL, HAS, WBL analyzed and interpreted the data and contributed to the manuscript.
The authors would like to thank all the patients with pyruvate kinase deficiency and their family members who contributed data to this natural history study.
For their work on the molecular analysis, the authors thank Paola Bianchi, Elisa Fermo, Patrick Gallagher, and Kimberly Lezon-Geyda.
RFG, HMY, JAR, EJN, WB, BG, SWE, EJvB, KK, YR, SC, JLK are scientific advisors to Agios Pharmaceuticals.
RFG, EJvB, BG, HMY, DHM, KK, JLK, YR, SS, and WB receive research funds from Agios Pharmaceuticals.
The remaining authors declare no competing financial interests.
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Research Funding:
The Pyruvate Kinase Deficiency Natural History Study was supported by research funding from Agios Pharmaceuticals.
Keywords:
- Science & Technology
- Life Sciences & Biomedicine
- Hematology
- THALASSEMIA PATIENTS
- DEFERASIROX
- CHELATION
- THERAPY
Prevalence and management of iron overload in pyruvate kinase deficiency: report from the Pyruvate Kinase Deficiency Natural History Study
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© 2019 Ferrata Storti Foundation
