Hemoglobinopathies
News & Literature Highlights
March 2022
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Hemoglobinopathies
Sustained Improvements in Patient-Reported Quality of Life Up to 24 Months Post-Treatment With LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy
ASH 2021 Annual Meeting and Exposition
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Hemoglobinopathies
Betibeglogene Autotemcel Gene Therapy for Non–β0/β0 Genotype β-Thalassemia
New England Journal of Medicine
February 2022
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Hemoglobinopathies
Luspatercept Improves Quality of Life and Reduces Red Blood Cell Transfusion Burden in Patients with Non-Transfusion-Dependent β-Thalassemia in the BEYOND Trial
ASH 2021 Annual Meeting and Exposition
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Hemoglobinopathies
ASH President Applauds Introduction of Legislation to Establish Sickle Cell Disease Medicaid Demonstration Program
American Society of Hematology
January 2022
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Hemoglobinopathies
Restoring Iron Homeostasis in Pts Who Achieved Transfusion Independence After Treatment With Betibeglogene Autotemcel Gene Therapy: Results From Up to 7 Years of Follow-Up
ASH 2021 Annual Meeting and Exposition
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Hemoglobinopathies
Mitapivat (AG-348) Demonstrates Safety, Tolerability, and Improvements in Anemia, Hemolysis, Oxygen Affinity, and Hemoglobin S Polymerization Kinetics in Adults With Sickle Cell Disease: A Phase 1 Dose Escalation Study
ASH 2021 Annual Meeting and Exposition
December 2021
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Hemoglobinopathies
Significance of Genetic Modifiers of Hemoglobinopathies Leading Towards Precision Medicine
Scientific Reports (Nature)
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Hemoglobinopathies
Extended Phenotyping Does Not Preclude the Occurrence of Delayed Haemolytic Transfusion Reactions in Sickle Cell Disease
British Journal of Haematology
November 2021
October 2021
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Hemoglobinopathies
Developing an Emergency Department Order Set to Treat Acute Pain in Sickle Cell Disease
Journal of the American College of Emergency Physicians Open (JACEP Open)
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Hemoglobinopathies
Manifestations of HbSE Sickle Cell Disease: A Systematic Review
Journal of Translational Medicine
September 2021
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Hemoglobinopathies
Techniques for the Detection of Sickle Cell Disease: A Review
Micromachines (Basel)
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Hemoglobinopathies
Impact of Hemoglobin Biophysical Studies on Molecular Pathogenesis and Drug Therapy for Sickle Cell Disease
Molecular Aspects of Medicine
August 2021
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Hemoglobinopathies
Fixing the Sickle Cell Disease Gene
NIH Research Matters
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Hemoglobinopathies
Neutrophil Binding on E-selectin: A Potential Biomarker of Clinical Course and Response to Therapy in Sickle Cell Disease
International Society on Thrombosis and Haemostasis 2021
July 2021
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Hemoglobinopathies
Luspatercept Safe, Effective in Nontransfusion-Dependent Beta-Thalassemia
HemOnc Today [Healio]
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Hemoglobinopathies
Cost-Effectiveness of a Hypothetical Cell or Gene Therapy Cure for Sickle Cell Disease
Scientific Reports [Nature Research]
June 2021
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Hemoglobinopathies
Impact of Renal Function on Hydroxyurea Exposure in Sickle-Cell Disease Patients
British Journal of Clinical Pharmacology
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Hemoglobinopathies
Effect of Poloxamer 188 vs Placebo on Painful Vaso-Occlusive Episodes in Children and Adults With Sickle Cell Disease: A Randomized Clinical Trial
JAMA
May 2021
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Hemoglobinopathies
Cancer Diagnosis in Patient Given LentiGlobin Revised to Severe Anemia
Sickle Cell Disease News
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Hemoglobinopathies
A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease
New England Journal of Medicine
April 2021
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Hemoglobinopathies
Usefulness of NGS for Diagnosis of Dominant Beta-Thalassemia and Unstable Hemoglobinopathies in Five Clinical Cases
Frontiers in Physiology
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Hemoglobinopathies
EMA Launches Safety Review After Gene Therapy Trial Patient Develops Blood Cancer
BioSpace
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Hemoglobinopathies
What Are the Key Considerations When Prescribing Pharmacotherapy for Sickle Cell Anemia?
Expert Opinion on Pharmacotherapy
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Hemoglobinopathies
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia
New England Journal of Medicine
