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The gene therapy is indicated for the treatment of children and adults with AADC deficiency, including the full spectrum of disease severity.
November 14, 2024
By: Charlie Sternberg
Associate Editor
The U.S. Food and Drug Administration (FDA) has approved PTC Therapeutics Inc.’s gene therapy for the treatment of AADC deficiency, making it the first-ever gene therapy approved in the United States that is directly administered to the brain. “PTC has once again pioneered a new approach to treating highly morbid neurologic diseases,” said Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics. “I am proud of our team’s unwavering commitment to achieve this...
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