Ptc Therapeutics
Clinical trials sponsored by Ptc Therapeutics, explained in plain language.
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Brain surgery delivers gene therapy to children with rare neurological disorder
Disease control OngoingThis study is testing the safety of a new surgical tool and a one-time gene therapy for children with Aromatic L-amino Acid Decarboxylase (AADC) deficiency, a rare genetic disorder that severely affects brain development and movement. The therapy, called eladocagene exuparvovec, …
Phase: PHASE2 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Apr 02, 2026 02:26 UTC
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Extended hope for rare neurological disease
Disease control OngoingThis study aims to see if the drug vatiquinone is safe and effective for people with Friedreich ataxia when taken for up to three years. It is for 130 participants who have already been taking vatiquinone in a previous trial. The main goal is to monitor for side effects while als…
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Mar 30, 2026 14:29 UTC
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New PKU treatment could ease strict diet rules
Disease control OngoingThis study is testing the long-term safety of an investigational medication called PTC923 for people with phenylketonuria (PKU). It involves 200 participants and aims to see if the treatment allows people to safely eat more protein while keeping their blood phenylalanine levels i…
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Mar 23, 2026 15:16 UTC