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  • PTC Therapeutics completes acquisition of ST-920 Fabry disease gene therapySep 22, 2026
  • PTC Therapeutics reported new Sephience data reinforcing broad patient benefits, including sustained metabolic control, greater dietary flexibility, and a consistent favorable safety profileAug 24, 2026
  • PTC announced plans to expand its rare disease portfolio through the acquisition of the BLA-stage ST-920 Fabry disease programAug 13, 2026
  • PTC Therapeutics reported positive topline results from the 24-month interim analysis of the PIVOT-HD extension study of VotoplamApr 29, 2026
  • PTC Therapeutics announces approval of Sephience™ (sepiapterin) for PKU treatment in JapanDec 22, 2025

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