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News about "PTC Therapeutics"
PTC Therapeutics Secures US FDA Approval for SEPHIENCE
PTC Therapeutics has received FDA approval for SEPHIENCE (sepiapterin) to treat sepiapterin-responsive phenylketonuria (PKU) in patients aged one month and older, offering a new therapy for managing this rare metabolic disorder.
PTC Therapeutics | 29/07/2025 | By Mrinmoy Dey | 129
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 | 195
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 | 655
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