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3. Diagnosis and management of pyruvate kinase deficiency: international expert guidelines

7. Clinically meaningful improvements in patient‐reported outcomes in mitapivat‐treated patients with pyruvate kinase deficiency

10. Osmotic gradient ektacytometry - a novel diagnostic approach for neuroacanthocytosis syndromes.

11. Comorbidities and complications in adult and paediatric patients with pyruvate kinase deficiency: Analysis from the Peak Registry.

12. Oxygen gradient ektacytometry-derived biomarkers are associated with acute complications in sickle cell disease

13. A novel composition of endogenous metabolic modulators improves red blood cell properties in sickle cell disease

14. Oxygen gradient ektacytometry-derived biomarkers are associated with acute complications in sickle cell disease

15. Metabolic blood profile and response to treatment with the pyruvate kinase activator mitapivat in patients with sickle cell disease.

17. Use of the oxygen gradient ektacytometry in the dose titration of hydroxyurea therapy in children with sickle cell disease.

19. A novel missense variant in ATP11C is associated with reduced red blood cell phosphatidylserine flippase activity and mild hereditary hemolytic anemia

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

23. The Pyruvate Kinase Deficiency Global Longitudinal (Peak) Registry: rationale and study design

24. Challenges and Opportunities of Precision Medicine in Sickle Cell Disease: Novel European Approach by GenoMed4All Consortium and ERN-EuroBloodNet

25. The Pyruvate Kinase Deficiency Global Longitudinal (Peak) Registry:rationale and study design

26. A novel missense variant in ATP11C is associated with reduced red blood cell phosphatidylserine flippase activity and mild hereditary hemolytic anemia

27. Activation of pyruvate kinase as therapeutic option for rare hemolytic anemias:Shedding new light on an old enzyme

28. A novel missense variant in ATP11C is associated with reduced red blood cell phosphatidylserine flippase activity and mild hereditary hemolytic anemia

29. One-year safety and efficacy of mitapivat in sickle cell disease: follow-up results of a phase 2, open-label study

30. Activation of pyruvate kinase as therapeutic option for rare hemolytic anemias: Shedding new light on an old enzyme

31. The Pyruvate Kinase Deficiency Global Longitudinal (Peak) Registry: rationale and study design

32. Early-onset reduced bone mineral density in patients with pyruvate kinase deficiency

33. Challenges and Opportunities of Precision Medicine in Sickle Cell Disease: Novel European Approach by GenoMed4All Consortium and ERN-EuroBloodNet

34. Heterozygosity for bisphosphoglycerate mutase deficiency expressing clinically as congenital erythrocytosis: A case series and literature review

35. A novel missense variant in $ATP11C$ is associated with reduced red blood cell phosphatidylserine flippase activity and mild hereditary hemolytic anemia

36. Early-onset reduced bone mineral density in patients with pyruvate kinase deficiency

37. Early‐onset reduced bone mineral density in patients with pyruvate kinase deficiency

39. Untargeted Metabolomics on Dried Blood Spots of Patients with Sickle Cell Disease Treated with the Pyruvate Kinase Activator Mitapivat

40. Age of Onset of Complications in Patients with Pyruvate Kinase Deficiency: Analysis from the Peak Registry

41. Comorbidities and Complications in Pediatric Patients with Pyruvate Kinase Deficiency Enrolled in the Peak Registry

42. An Endogenous Metabolic Modulator Composition Improves Hydration Status and Decreases Adhesion Properties of Red Blood Cells from Patients with Sickle Cell Disease

44. The Launch of Two Sub-Studies of the Peak Registry, a Global, Longitudinal Study of Pyruvate Kinase Deficiency

45. Mitapivat Improves Iron Overload in Patients with Pyruvate Kinase Deficiency

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

47. Long-Term Hemoglobin Response and Reduction in Transfusion Burden Are Maintained in Patients with Pyruvate Kinase Deficiency Treated with Mitapivat

49. Heterozygosity for bisphosphoglycerate mutase deficiency expressing clinically as congenital erythrocytosis: A case series and literature review

50. Transfusion burden in early childhood plays an important role in iron overload in Diamond‐Blackfan anaemia

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