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PTC Therapeutics' Rare Genetic Disease Gene Therapy Shows Durable Improvement Even At 5 Years

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  • PTC Therapeutics Inc (NASDAQ: PTCT) presented a new analysis of five-year results of gene therapy, PTC-AADC.

  • The data exhibited improvements in children with aromatic L-Amino acid decarboxylase (AADC) deficiency.

  • AADC deficiency is a rare genetic disorder that causes severe disability.

  • Children treated with PTC-AADC developed motor function and cognitive skills not previously seen, such as holding up their head, sitting or standing with support, and communicating. These persisted for up to 10 years.

  • In addition, the rate of respiratory infection declined from an average of 2.4 episodes per year at 12 months to 0.6 episodes per year at 2 years and 0.3 episodes per year at 5 years.

  • Almost all treated children went from a baseline weight below the third percentile to making age-appropriate weight gains by 12 months following gene therapy treatment.

  • PTC-AADC is currently under review by the European Medicines Agency's Committee for Medicinal Products for Human Use, with an opinion expected in Q4 of 2021.

  • A Biologics License Application is expected to be submitted to the FDA by the end of 2021.

  • Related Link: PTC Therapeutics/Roche's Evrysdi Approved In Europe For Muscular Atrophy.

  • Price Action: PTCT stock is down 1.37% at $37.77 during the market session on the last check Wednesday.

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