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Nov 28-29, 2024 Dubai, UAE

International Conference on

Rare Diseases and Orphan Drugs

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Hemophilia A Gene Therapy Displays Superiority in Phase 3 AFFINE Trial

2024-07-24 - 2024-07

Topline data from the Phase 3 AFFINE study demonstrate the potential advantage of giroctocogene fitelparvovec, an investigational one-time gene therapy, for the treatment of adults with moderately severe to severe hemophilia A.1 Announced by Pfizer on July 24, 2024, giroctocogene fitelparvovec met the primary non-inferiority and key superiority objectives of total annualized bleeding rate (ABR) in a 15-month follow-up period compared with standard-of-care Factor VIII (FVIII) replacement prophylaxis.

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Hemophilia A Gene Therapy Displays Superiority in Phase 3 AFFINE Trial

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