408 results on '"van Beers"'
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2. Prediction of high- and low-risk multiple myeloma based on gene expression and the International Staging System
3. Long-Term Improvements in Patient-Reported Outcomes in Patients with Pyruvate Kinase Deficiency Treated with Mitapivat
4. Comorbidities and Complications in Pediatric Patients with Pyruvate Kinase Deficiency Enrolled in the Peak Registry
5. Age of Onset of Complications in Patients with Pyruvate Kinase Deficiency: Analysis from the Peak Registry
6. Mitapivat Improves Iron Overload in Patients with Pyruvate Kinase Deficiency
7. Long-Term Hemoglobin Response and Reduction in Transfusion Burden Are Maintained in Patients with Pyruvate Kinase Deficiency Treated with Mitapivat
8. Untargeted Metabolomics on Dried Blood Spots of Patients with Sickle Cell Disease Treated with the Pyruvate Kinase Activator Mitapivat
9. An Endogenous Metabolic Modulator Composition Improves Hydration Status and Decreases Adhesion Properties of Red Blood Cells from Patients with Sickle Cell Disease
10. Mitapivat Improves Markers of Hemolysis and Erythropoiesis in Patients with Pyruvate Kinase Deficiency Irrespective of Hemoglobin Response
11. Untargeted Metabolomics on Dried Blood Spots of Patients with Sickle Cell Disease Treated with the Pyruvate Kinase Activator Mitapivat
12. Age of Onset of Complications in Patients with Pyruvate Kinase Deficiency: Analysis from the Peak Registry
13. Comorbidities and Complications in Pediatric Patients with Pyruvate Kinase Deficiency Enrolled in the Peak Registry
14. An Endogenous Metabolic Modulator Composition Improves Hydration Status and Decreases Adhesion Properties of Red Blood Cells from Patients with Sickle Cell Disease
15. Movement Behavior in Adults with Sickle Cell Disease Compared to Healthy Adults: Preliminary Results
16. The Launch of Two Sub-Studies of the Peak Registry, a Global, Longitudinal Study of Pyruvate Kinase Deficiency
17. Mitapivat Improves Iron Overload in Patients with Pyruvate Kinase Deficiency
18. Long-Term Improvements in Patient-Reported Outcomes in Patients with Pyruvate Kinase Deficiency Treated with Mitapivat
19. Long-Term Hemoglobin Response and Reduction in Transfusion Burden Are Maintained in Patients with Pyruvate Kinase Deficiency Treated with Mitapivat
20. A remarkable case of HbH disease illustrates the relative contributions of the α-globin enhancers to gene expression
21. Interplay of erythropoietin, fibroblast growth factor 23, and erythroferrone in patients with hereditary hemolytic anemia
22. The Launch of Two Sub-Studies of the Peak Registry, a Global, Longitudinal Study of Pyruvate Kinase Deficiency
23. Mitapivat Improves Markers of Hemolysis and Erythropoiesis in Patients with Pyruvate Kinase Deficiency Irrespective of Hemoglobin Response
24. Movement Behavior in Adults with Sickle Cell Disease Compared to Healthy Adults: Preliminary Results
25. Early-Onset Osteopenia and Osteoporosis in Patients with Pyruvate Kinase Deficiency
26. Decreased activity and stability of pyruvate kinase in sickle cell disease: a novel target for mitapivat therapy
27. Cerebrovascular reserve capacity is impaired in patients with sickle cell disease
28. Ferritin Levels Do Not Reflect the Severity of Iron Overload in Diamond Blackfan Anemia
29. Pharmacodynamic Effects of AG-946, a Highly Potent Next-Generation Activator of Pyruvate Kinase, in Ex Vivo Treatment of Red Blood Cells from Sickle Cell Disease Patients
30. A Phase 2/3, Randomized, Double-Blind, Placebo-Controlled Study of Mitapivat in Patients with Sickle Cell Disease
31. Safety and Efficacy of Mitapivat (AG-348), an Oral Activator of Pyruvate Kinase R, in Subjects with Sickle Cell Disease: A Phase 2, Open-Label Study (ESTIMATE)
32. Characterizing Iron Overload By Age in Patients Diagnosed with Pyruvate Kinase Deficiency - a Descriptive Analysis from the Peak Registry
33. A Novel Quali-Quantitative Defect of VWF
34. Durability of Hemoglobin Response and Reduction in Transfusion Burden Is Maintained over Time in Patients with Pyruvate Kinase Deficiency Treated with Mitapivat in a Long-Term Extension Study
35. Identification of Biomarkers That Are Associated with Clinical Complications of Hemoglobin SC Disease and Sickle Cell Anemia
36. Bone Mineral Density Remains Stable in Pyruvate Kinase Deficiency Patients Receiving Long-Term Treatment with Mitapivat
37. Mitapivat Improves Ineffective Erythropoiesis and Reduces Iron Overload in Patients with Pyruvate Kinase Deficiency
38. One-Year Safety and Efficacy of Mitapivat in Sickle Cell Disease: Follow-up Results of a Phase 2, Open-Label Study
39. Roxyscan: A Novel Method to Assess Red Blood Cell Resilience to Oxidative Stress in Sickle Cell Disease
40. Novel Biomarkers for Assessing Clinical Severity in Hereditary Spherocytosis - Application of Routine and Advanced Diagnostic Tests
41. Improvements in Markers of Hemolysis and Liver Iron Concentration in Mitapivat-Treated Adult Patients with a Delayed Hemoglobin Response
42. Regional Genetic Heterogeneity Among Patients with Pyruvate Kinase Deficiency
43. Safety and Efficacy of Mitapivat in Adult Patients with Erythrocyte Membranopathies (SATISFY)
44. Pyruvate Kinase Thermostability Is Associated with Red Blood Cell Adhesion, Deformability and Oxygen Affinity in Patients with Sickle Cell Disease
45. A remarkable case of HbH disease illustrates the relative contributions of the α-globin enhancers to gene expression
46. The Prognostic Power of Gene Expression Profiling with Cytogentics and Routinely Acquired Serum Markers: SKY92 Combined with Revised ISS
47. Early-Onset Osteopenia and Osteoporosis in Patients with Pyruvate Kinase Deficiency
48. Baseline Characteristics of Patients in Peak: A Global, Longitudinal Registry of Patients with Pyruvate Kinase Deficiency
49. Red Cell Rheology Biomarkers to Assess Cure in Gene-Based Therapies
50. Oxygen Gradient Ektacytometry-Derived Biomarkers Are Associated with the Occurrence of Cerebral Infarction, Acute Chest Syndrome and Vaso-Occlusive Crisis in Sickle Cell Disease
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