Impact of idebenone on pulmonary morbidity, including bronchopulmonary adverse events, in Duchenne muscular dystrophy (DMD)

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In DMD, respiratory function declines due to progressive muscle weakness, with the decline accelerating once patients become non-ambulatory. As well-described clinical thresholds are crossed (based on forced vital capacity, percentage of predicted value; FVC%p), the risk and severity of respiratory complications, including bronchopulmonary adverse events (BAEs) and sleep disturbances, increases, causing early morbidity. In the 52 week, Phase 3 clinical trial in DMD (DELOS), idebenone significantly slowed respiratory function decline.

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