Effects of the simultaneous administration of diethylstilbestrol and prednisolone on serum enzymes in Duchenne's muscular dystrophy.

The effects of diethylstilbestrol (DES) and prednisolone (Pr), administered alone or in combination, on the serum enzyme activities in Duchenne's muscular dystrophy (DMD) have been evaluated. When DES and Pr were given simultaneously to four boys with DMD, the mean creatine phosphokinase and lactate dehydrogenase levels fell to about one half those achieved with either agent alone. No untoward or unexpected side effects were encountered. These observations, and our previous demonstration that both agents reduce enzyme efflux from skeletal muscle, give promise that a safe combination therapy may be found to approximately normalize the serum in DMD. This will permit us to test the hypothesis that lowering the serum enzymes, by reducing their loss from skeletal muscle, will slow or arrest the muscle deterioration characteristic of this disease.