Ptc Therapeutics
Clinical trials sponsored by Ptc Therapeutics, explained in plain language.
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New PKU drug could loosen strict diet restrictions
Disease control OngoingThis phase 3 study tests a drug called PTC923 (sepiapterin) in 200 people with phenylketonuria (PKU), a genetic condition that requires a strict low-protein diet. The main goal is to see if the drug is safe over the long term and whether it allows people to eat more protein witho…
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 14:03 UTC
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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: PHASE2 • 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: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 07:56 UTC