Prednisone treatment in Duchenne muscular dystrophy. Long-term benefit.

We report the long-term results of a therapeutic trial of prednisone in the treatment of Duchenne muscular dystrophy, comparing the age at which 16 treated patients and 38 controls lost the ability to ambulate. Survival curve analysis and group mean comparisons indicate that ambulation was significantly prolonged, by approximately two years, in the prednisone-treated group compared with the control group. The results of this study indicate that prednisone therapy has a long-term beneficial effect in the treatment of Duchenne muscular dystrophy, in addition to the short-term palliation we previously reported. Further studies are needed to establish optimal treatment schedules using prednisone and to develop improved therapeutic agents.