Luspatercept as Potential Treatment for Congenital Sideroblastic Anemia
Author:
Affiliation:
1. Erasmus University Medical Center, Rotterdam, the Netherlands
Publisher
Massachusetts Medical Society
Subject
General Medicine
Link
http://www.nejm.org/doi/pdf/10.1056/NEJMc2216213
Reference4 articles.
1. Understanding Sideroblastic Anemia: An Overview of Genetics, Epidemiology, Pathophysiology and Current Therapeutic Options
Understanding Sideroblastic Anemia: An Overview of Genetics, Epidemiology, Pathophysiology and Current Therapeutic Options
2. Luspatercept in Patients with Lower-Risk Myelodysplastic Syndromes
3. A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent β-Thalassemia
4. Sideroblastic anemia: molecular analysis of the ALAS2 gene in a series of 29 probands and functional studies of 10 missense mutations
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1. A novel and apparent de novo ALAS2 missense variant associated with congenital sideroblastic anemia;Frontiers in Pediatrics;2024-08-29
2. Severe pulmonary arterial hypertension in congenital sideroblastic anemia from PUS1 mutation – a case report;BMC Medical Genomics;2024-08-15
3. Luspatercept enhances hemoglobin levels in a Chinese boy with congenital sideroblastic anemia: A case report;World Journal of Clinical Cases;2024-07-06
4. Luspatercept for the treatment of congenital sideroblastic anemia: Two case reports;Current Research in Translational Medicine;2024-03
5. A novel ALAS2 mutation causes congenital sideroblastic anemia;Mediterranean Journal of Hematology and Infectious Diseases;2023-10-30
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