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Idebenone as a novel therapeutic approach for Duchenne Muscular Dystrophy


Buyse, GM and Gueven, N and McDonald, CM, Idebenone as a novel therapeutic approach for Duchenne Muscular Dystrophy, European Neurological Review, 10, (2) pp. 189-94. ISSN 1758-3837 (2015) [Refereed Article]


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DOI: doi:10.17925/ENR.2015.10.02.189


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.

Item Details

Item Type:Refereed Article
Keywords:Duchenne Muscular Dystrophy, idebenone, respiratory function, peak expiratory flow, glucocorticoid steroid
Research Division:Biomedical and Clinical Sciences
Research Group:Clinical sciences
Research Field:Clinical sciences not elsewhere classified
Objective Division:Expanding Knowledge
Objective Group:Expanding knowledge
Objective Field:Expanding knowledge in the health sciences
UTAS Author:Gueven, N (Dr Nuri Guven)
ID Code:106147
Year Published:2015
Deposited By:Pharmacy
Deposited On:2016-02-01
Last Modified:2016-09-09
Downloads:176 View Download Statistics

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