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Department of Neurology, Washington University School of Medicine, St. Louis, MO (Drs. Brooke and Gilder, and J. Florence, J. Schierbecker, and K.K. Kaiser)
Department of Biostatistics, Washington University School of Medicine, St. Louis, MO (J.P. Miller, S. Mandel, and Dr. Arfken)
Departments of Neurology, Vanderbilt University, Nashville, TN (Dr. Fenichel and J. Robison)
Ohio State University, Columbus, OH (Dr. Mendell, and W.M. King and L. Signore)
University of Rochester, Rochester, NY (Drs. Griggs and Moxley, and S. Pandya).
Two-hundred eighty-three boys with Duchenne dystrophy and 10 with Becker dystrophy have been followed for up to 10 years in a protocol that accurately measured their function, strength, contractures, and back curvature. Clinical heterogeneity is noted. Patients whose muscles were stronger were more likely to die from a cardiomyopathy. Weaker patients died from respiratory failure. A series of milestones is defined, which is of use in following the illness in an individual patient. This approach permits a scoring system that allows the severity of the disease to be defined in an individual boy. Evaluation of physical therapy and surgical intervention shows that night splints and scoliosis surgery are effective forms of treatment.
Address correspondence and reprint requests to Dr. Brooke, Division of Neurology, Room 2E3.12, Walter C. McKenzie Center Health Sciences, Edmonton, Alberta, Canada T6G 2R3.
Supported by a grant from the Muscular Dystrophy Association and Public Health Research Grant No. RR-36 from the General Clinical Research Center Branch, Division of Research Facilities and Resources, Bethesda, MD.
Received September 7, 1988. Accepted for publication in final form October 24, 1988.
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