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The genetic defect causes problems with coordination and balance, muscle control and weakness, vision impairment, hearing problems, scoliosis, diabetes and others. The deficiency of frataxin causes iron accumulation in mitochondria and oxidative stress, which damages nerve cells (neurons), especially in the spinal cord and brain.

The global Friedreich's ataxia market is estimated to be valued at US$ 1121.42 Mn in 2024 and is expected to exhibit a CAGR of 13.% over the forecast period 2024 to 2031.

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