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Your search keyword '"Friedreich ataxia"' showing total 1 results
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1. PTC Therapeutics Announces Vatiquinone NDA Submission to FDA for the Treatment of Children and Adults Living with Friedreich Ataxia.

Abstract

PTC Therapeutics has submitted an NDA to the FDA for vatiquinone, a potential treatment for Friedreich ataxia in children and adults. The submission is based on data from clinical studies showing significant and durable evidence of slowing disease progression and safety across all age groups. This marks the fourth approval application PTC has submitted to the FDA in 2025, following successful approval of their AADC gene therapy. [Extracted from the article]

Published
2025

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