PTC Therapeutics Inc. has shared its corporate presentation highlighting key achievements and regulatory milestones. In 2024, the company successfully submitted four U.S. regulatory approval applications, securing approval for the AADC Gene Therapy BLA and Sephience (sepiapterin) PKU NDA. Additionally, the Translarna nmDMD NDA and Vatiquinone Friedreich's Ataxia NDA were accepted. These accomplishments in 2024 have set a strong foundation for the company's continued success in 2025 and beyond. PTC Therapeutics emphasizes its focus on developing transformative therapies for patients with rare diseases. You can access the full presentation through the link below.
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