Proposed treatment of Duchenne muscular dystrophy with desferrioxamine.

The primary disturbance in Duchenne muscular dystrophy (DMD) appears to affect membrane function, and changes characteristic of oxidant-induced damage occur in skeletal muscle and erythrocytes. There is recent evidence that DMD is a functional tocopherol deficiency, with reduced levels of the lipoprotein required to carry tocopherol to tissues. This may explain the parallels between DMD and dietary tocopherol deficiency. Thus DMD should follow the usual experience of other examples of oxidative pathology, where the balance between tocopherol, the main antioxidant in membrane lipids, and non protein-bound iron, an important catalyst of reactions which produce oxidizing free radicals, largely determines whether or not tissue damage occurs. Desferrioxamine prevents oxidant damage in vitro and in vivo by removing this iron, and may therefore be able to reverse the muscle damage of DMD. Recent experience with this drug in long term dialysis patients is consistent with this suggestion.