Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trial

@article{Buyse2015EfficacyOI,
  title={Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trial},
  author={Gunnar M. Buyse and Thomas Voit and Ulrike Schara and Chiara S. M. Straathof and Maria Grazia D'angelo and Guenther Bernert and J M Cuisset and Richard S. Finkel and Nathalie M. Goemans and Craig M. McDonald and Christian Rummey and Thomas Meier},
  journal={The Lancet},
  year={2015},
  volume={385},
  pages={1748-1757}
}
BACKGROUND Cardiorespiratory failure is the leading cause of death in Duchenne muscular dystrophy. Based on preclinical and phase 2 evidence, we assessed the efficacy and safety of idebenone in young patients with Duchenne muscular dystrophy who were not taking concomitant glucocorticoids. METHODS In a multicentre phase 3 trial in Belgium, Germany, the Netherlands, Switzerland, France, Sweden, Austria, Italy, Spain, and the USA, patients (age 10-18 years old) with Duchenne muscular dystrophy… CONTINUE READING
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