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


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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