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Long-Term Benefits Put PTC Friedreich’s Ataxia Drug Back On Track

 
• By Kevin Grogan

While vatiquinone may have missed its primary endpoint in a Phase III trial, PTC is ready to file the drug for the debilitating, life-shortening disorder after showing that it slowed disease progression over 144 weeks.

Frataxin
FA is usually caused by a single genetic defect that leads to reduced production of frataxin • Source: Shutterstock

The development road has been a rocky one for PTC Therapeutics, Inc.'s vatiquinone but the company has unveiled long-term data which will provide support for a regulatory filing in the rare neuromuscular disorder Friedreich’s ataxia (FA).

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