PTC Therapeutics reports 50% slowdown in Friedreich ataxia progression with vatiquinone treatment in MOVE-FA study.

PTC Therapeutics reported positive long-term results for vatiquinone, a treatment for Friedreich ataxia (FA), a rare genetic disorder. In the MOVE-FA study, patients showed a 3.7-point improvement on the modified Friedreich Ataxia Rating Scale (mFARS) after 144 weeks, indicating a 50% slowdown in disease progression. The company intends to submit a New Drug Application to the FDA in December. Following the news, PTC's shares rose over 5% in pre-market trading.

October 08, 2024
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