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FDA Approves First-Ever Brain-Injected Gene Therapy for AADC Deficiency

November 19, 2024 The U.S. Food and Drug Administration (FDA) has granted fast-track approval to Kebilidi (Eladocagene exuparvovec), the first gene therapy in the United States administered via direct intracerebral injection. This approval represents a significant advance in the…

Michael V Johnston [1946 - 2022]

I am deeply saddened to learn that Michael V Johnston MD. Professor Emeritus of Pediatrics and Neurology at the Johns Hopkins University…

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