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New Review of Idebenone as a Novel Therapeutic Approach for Duchenne Muscular Dystrophy

LONDON, January 12, 2016 /PRNewswire/ --

European Neurological Review, the peer-reviewed journal, has published a review highlighting the promising studies of idebenone as a novel therapeutic approach for Duchenne muscular dystrophy



Progressive loss of pulmonary function leads to early morbidity and mortality in Duchenne muscular dystrophy (DMD) due to both expiratory impairment with ineffective airway clearance, and inspiratory impairment leading to nocturnal and daytime hypoventilation and respiratory failure. Glucocorticoid steroids have become a mainstay of DMD therapy with well-documented efficacy on muscle strength and respiratory function. However, the side-effect profile restricts their long-term use, particularly in non-ambulant patients. Idebenone improves secondary mitochondrial dysfunction caused by dystrophin deficiency, intracellular calcium accumulation and increased reactive oxygen species (ROS). Idebenone-mediated improved bioenergetics leads to enhanced adenosine triphosphate (ATP) production and reduced ROS. Based on this rationale, idebenone has been investigated clinically for efficacy on reducing respiratory function decline in exploratory phase II (DELPHI) and confirmatory phase III (DELOS) trials. Idebenone significantly reduced the loss of respiratory function in 8-18-year-old DMD patients who were not using concomitant glucocorticoids. These results indicate that idebenone can modify the natural course of respiratory disease progression in DMD, which is relevant in clinical practice where loss of respiratory function continues to be a predominant cause of early morbidity and mortality in DMD.

The full peer-reviewed, open-access article is available here:

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European Neurological Review, a peer-reviewed, open access, bi-annual journals specialising in the publication of balanced and comprehensive review articles written by leading authorities to address the most important and salient developments in the field of neurology. The aim of these reviews is to break down the high science from 'data-rich' primary papers and provide practical advice and opinion on how this information can help physicians in the day to day clinical setting. Practice guidelines, symposium write-ups, case reports, and original research articles are also featured to promote discussion and learning amongst physicians, clinicians, researchers and related healthcare professionals.

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