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VisitHow many patients will be treated with PTC's AADC gene therapy by end of 2025?
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PTC Therapeutics' annual reports and press releases
FDA Approves PTC Therapeutics' Gene Therapy for AADC Deficiency
Nov 13, 2024, 11:18 PM
The U.S. Food and Drug Administration (FDA) has approved PTC Therapeutics' gene therapy for Aromatic L-Amino Acid Decarboxylase (AADC) deficiency, a potentially fatal enzyme deficiency disorder. This therapy is administered directly in the brain, marking a significant advancement in the treatment of neurological conditions. The approval has positively impacted PTC Therapeutics' stock, which saw a 2% increase in aftermarket trading.
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