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SCDAA Statement About Gene Therapy Approval

On Dec. 8, 2023, the Food and Drug Administration (FDA) approved two cell-based gene therapies for sickle cell disease (SCD), Casgevy from CRISPR/Vertex and Lyfgenia from bluebird bio. These are the first treatments of their kind available to individuals with SCD in the United States. SCDAA welcomes the approval of these potentially curative therapies which mark major advances in the treatment of sickle cell disease; however, there are valid concerns about accessibility and the potential for adverse effects.

Source: https://www.sicklecelldisease.org/2023/12/08/scdaa-statement-about-gene-therapy-approval/

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SCDAA Statement About Gene Therapy Approval

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