STEROID THERAPY IN DUCHENNE MUSCULAR DYSTROPHY (DMD)

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Prospective/Randomized Multi-Institution: 13 CINRG centers Subject Population: Ambulant boys aged 4-10 years with established DMD diagnosis, naive to treatment with glucocorticoids. Number of Subjects: 140 Treatment groups (35 patients per group) 4-<7 years of age daily prednisone 4-<7 years of age high-dose weekly prednisone 7+ to 10 years of age daily prednisone 7+ to 10 years of age high-dose weekly prednisone Primary Endpoints: Strength: Percent change in QMT scores of upper and lower extremities. Side-Effects: Difference in absolute means of the pre and post treatment change in height and weight/height ratio. Duration of Study: twelve (12) 28-day treatment cycles. (Approximately 12 months). Frequency of Follow-Ups: Visits at month 1 and month 3, then every 3 months.