PTC Therapeutics Submits NDA for Vatiquinone to FDA
The vatiquinone NDA is based on data from the placebo-controlled MOVE-FA study as well as results from two long-term studies including pediatric and adult FA patients.
PTC Therapeutics | 24/12/2024 | By Aishwarya
FDA Accepts PTC Therapeutics' Application for Upstaza Gene Therapy for AADC Deficiency
The BLA acceptance comes with a Priority Review designation, highlighting the urgent need for therapeutic options for patients suffering from AADC deficiency.
PTC Therapeutics | 15/05/2024 | By Abha
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