Company Overview - PTC Therapeutics is a global biopharmaceutical company focused on rare diseases, with a portfolio that includes six self-marketed products worldwide [4] - The company has a robust R&D pipeline, highlighted by its small molecule splicing platform, which has produced Evrysdi for spinal muscular atrophy (SMA) and Votoplam for Huntington's disease [4] Financial Performance - For the third quarter, PTC Therapeutics reported strong performance with revenues of $211 million, which included contributions from Sephience, their phenylketonuria (PKU) drug [5] - The company maintains a strong financial position with approximately $1.7 billion on its balance sheet [5] Product Launch - The launch of Sephience is a significant event for the company, providing a foundation for future growth [5]

PTC Therapeutics November 2025 Pam and Kelsey living with PKU This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this presentation, other than statements of historic fact, are forward-looking statements, including statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory subm ...