Maria Domenica Cappellini, MD, University of Milan, Milan, Italy, discusses the treatment landscape of beta thalassemia, focusing on exciting novel treatment options which have the potential to improve outcomes in patients with the disease in the future. This includes the CRISPR/Cas9 genome-editing therapy, which was recently approved for the treatment of sickle cell disease (SCD). This interview took place at the 65th ASH Annual Meeting and Exposition, held in San Diego, CA.
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Looking to the future in the treatment of beta thalassemia
Теги
Speaker: Maria Domenica CappelliniEvent: ASH 2023Institution: University of MilanFormat: InterviewSubject: ThalassemiaSubject: Non-MalignantField: TreatmentMedicines: LuspaterceptMedicines: Mitapivatbeta-thalassemiatransfusion-dependentnon-transfusion-dependentsickle cell diseasevaso-occlusive crisisVOCgene therapygene additionhemoglobinPK activityATP productiontransfusion burdenpyruvate kinase activatorFDA approvalField: Gene therapy