Duchenne Muscular Dystrophy as a mitochondrial myopathy: Why therapeutically targeting the mitochondria is a plausible treatment avenue

Rybalka, Emma ORCID: 0000-0002-4854-0036 and Timpani, Cara (2015) Duchenne Muscular Dystrophy as a mitochondrial myopathy: Why therapeutically targeting the mitochondria is a plausible treatment avenue. Project Report. Victoria University, Melbourne, Australia.

Abstract

More than 50 years of basic and clinical research highlights that gross metabolic impairment is an important yet often ignored feature of Duchenne Muscular Dystrophy-associated dystrophinopathy. We believe that mitochondrial dysfunction (specifically Complex I) is an aetiological modifier and promoter of the clinical progression of DMD, and that the mitochondria is a worthy candidate for therapeutic target. There is strong evidence that by-passing the Complex I deficit and stimulating Complex II (FADH2)/III-dependent energy production with oral Idebenone therapy is efficacious both in animal models and human DMD patients. There are other obvious benefits of Idebenone therapy that relate to its strong antioxidant potential and membrane protective effects. It is thus our opinion that Idebenone represents a novel and clinically relevant therapy for the treatment of a key aetiological modifier of DMD.

Additional Information

This publication constitutes an Expert Opinion Summary Report as part of Santhera Pharmaceutical's file for regulatory approvals in the USA and in Europe, for the treatment of Duchenne Muscular Dystrophy with Raxone(TM) (Idebenone).

Item type Monograph (Project Report)
URI https://vuir.vu.edu.au/id/eprint/30859
Subjects Current > FOR Classification > 1109 Neurosciences
Current > FOR Classification > 1115 Pharmacology and Pharmaceutical Sciences
Current > Division/Research > College of Health and Biomedicine
Keywords Duchenne Muscular Dystrophy; mitochondria; myopathy; Idebenone; metabolic therapy
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