Time to read: 03:35. Physicians may soon have a new tool for managing haemophilia A, as promising early results from an ongoing Phase 3 AFFINE study suggest that giroctocogene fitelparvovec, a one-time gene therapy, could become the second U.S. Food and Drug Administration (FDA)-approved gene therapy for this condition.

Source: Medscape, American Society of Hematology 2024

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Time to read: 02:46 minutes. The phase 3 study demonstrated that fidanacogene elaparvovec offers an effective benefit-risk profile, delivering efficacy at one of the lowest doses of AAV-based gene therapy for hemophilia B.

Source: NEJM

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