Duchenne muscular dystrophy: focus on pharmaceutical and nutritional interventions.
Duchenne muscular dystrophy is a lethal X-linked muscle disease resulting from a defect in the muscle membrane protein dystrophin. The absence of dystrophin leads to muscle membrane fragility, muscle death (necrosis) and eventual replacement of skeletal muscle by fat and fibrous connective tissue. Extensive muscle wasting and respiratory failure results in premature death often by the early 20s. This short review evaluates drug and nutritional interventions designed to reduce the severity of muscular dystrophy, while awaiting the outcome of research into therapies to correct the fundamental gene defect. Combinations of dietary supplementation with amino-acids such as creatine, specific anti-inflammatory drugs and perhaps drugs that target ion channels might have immediate realistic clinical benefits although rigorous research is required to determine optimal combinations of such interventions.
Radley
H G
HG
School of Anatomy and Human Biology, University of Western Australia, Crawley, Australia.
De Luca
A
A
Lynch
G S
GS
Grounds
M D
MD
D000305:Adrenal Cortex Hormones / Q000627:therapeutic use; D000318:Adrenergic beta-Agonists / Q000627:therapeutic use; D000818:Animals; D000893:Anti-Inflammatory Agents / Q000627:therapeutic use; D016207:Cytokines / Q000037:antagonists & inhibitors; D019587:Dietary Supplements; D006801:Humans; D007473:Ion Channels / Q000378:metabolism; D051379:Mice; D018101:Mice, Inbred mdx; D009137:Muscular Dystrophy, Animal / Q000178:diet therapy / Q000188:drug therapy; D020388:Muscular Dystrophy, Duchenne / Q000178:diet therapy* / Q000188:drug therapy*; D011480:Protease Inhibitors / Q000627:therapeutic use
D000305:Adrenal Cortex Hormones; D000318:Adrenergic beta-Agonists; D000893:Anti-Inflammatory Agents; D016207:Cytokines; D007473:Ion Channels; D011480:Protease Inhibitors