PTC Therapeutics
Clinical trials sponsored by PTC Therapeutics, explained in plain language.
A biopharmaceutical company focused on rare genetic disorders.
Not investment advice. Company information is shown for context only. Clinical trials frequently fail.
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Brain gene therapy delivery system tested in kids with rare disorder
Disease control OngoingThis study tests a special needle (SmartFlow cannula) designed to safely deliver a gene therapy drug (eladocagene exuparvovec) into the brains of 13 children with AADC deficiency, a rare genetic disorder that affects movement and development. The main goals are to check if the pr…
Phase 2 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 12:23 UTC
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Can a new drug slow friedreich ataxia? Long-Term trial underway
Disease control OngoingThis study is testing the long-term safety of a drug called vatiquinone in 130 people with Friedreich ataxia, a rare genetic disease that affects movement and coordination. All participants have taken vatiquinone before in earlier studies. Researchers will track side effects and …
Phase 3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 07:56 UTC