Vertex Presents New Data on CASGEVY®, Including First European Presentation of Data in Children Ages 5–11, at the European Hematology Association Congress and Announces Additional Global Regulatory Submissions
View original at finance.yahoo.comVertex Presents New Data on CASGEVY®, Including First European Presentation of Data in Children Ages 5–11, at the European Hematology Association Congress and Announces Additional Global Regulatory Submissions - Data from pivotal studies of CASGEVY in children ages 5–11 with severe sickle cell disease or transfusion-de…
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The data presented at EHA and published in NEJM underscore the consistent, durable and transformative benefits CASGEVY can provide to people living with sickle cell disease or transfusion-dependent beta thalassemia from early in life.
60% confidenceEfficacy and safety outcomes of CASGEVY in children ages 5–11 are consistent with the transformative profile established in adult and adolescent patients.
60% confidenceChildren living with sickle cell disease and transfusion-dependent beta thalassemia carry a significant disease burden from a very young age, with progressive complications leading to irreversible and life-shortening consequences, even with optimized supportive therapy.
60% confidenceThe CASGEVY pediatric data represent a profoundly important step forward, with the possibility of providing earlier intervention to prevent complications in children, for families who have had limited potentially curative options.
60% confidence