Friedreich's ataxia is a rare and debilitating neurodegenerative disorder caused by a mutation in the FXN gene, which encodes the protein frataxin. This condition is marked by various symptoms, such as coordination difficulties, a progressive decline in mobility, general weakness, sensory loss, scoliosis, diabetes, and cardiomyopathy, along with impaired vision, hearing, and speech. It is estimated that around 20,000 people worldwide are affected by Friedreich's ataxia, with approximately 5,000 in the United States and most of the rest living in Europe.
Reata Pharmaceuticals' Skyclarys, approved last February, is the first and only FDA-approved treatment for Friedreich's ataxia (FA). Reata was acquired by Biogen for approximately $7.3 billion last July.
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