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.