Preserving function in Duchenne dystrophy with long-term pulse prednisone therapy

We report a case of a patient with Duchenne muscular dystrophy who was trea ted with intermittent pulse prednisone for severe asthma from age 3 to 17 y r and had remarkable preservation of skeletal muscle function. He had a mat ernal uncle with Duchenne muscular dystrophy who had the identical familial deletion mutation and died at age 19 of respiratory failure. Compared with his untreated uncle, our patient remains partially ambulatory at age 20. T his case provides interesting, albeit anecdotal, evidence of considerable c linical benefit from pulse prednisone used on a much longer term basis than has been previously studied and promotes the need for further investigatio n on this type of therapy in Duchenne muscular dystrophy.