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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]

A patent has been issued for materials and methods to treat Friedreich's Ataxia, a rare genetic disorder that causes progressive neurological and cardiac dysfunction. The patent describes a new fusion polypeptide that aims to overcome limitations of existing treatments by providing higher solubility and allowing for higher doses to be administered. This development addresses the urgent need for new therapies to treat Friedreich's Ataxia, which currently has no cure. [Extracted from the article]