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Regression analyses accounted for baseline characteristics, baseline medical care consumption, and follow-up duration in a 1:1 matched sample (283 for each group matched on age, disease progression stage, and medical events during baseline) and in a full sample analysis (314 treated and 648 control). Treated pts on average were 14.1 years old at initiation. Eteplirsen claims were observed for a median of 13.0 months. Median follow-up was 27.3 months. Pts without eteplirsen claims in later months may have continued to receive treatment via the manufacturer's patient support program. Significant treatment effects of eteplirsen were observed on yearly average rates of emergency room visits (3.05 vs 5.46, P = 0.031), pulmonary management visits (1.56 vs 2.44, P = 0.039), and tracheostomy (0.25 vs 1.13, P = 0.015). Consistent results were also found in the full sample analysis (2.61 vs 4.67, p = 0.010 for emergency room visits; 2.14 vs 3.00, P = 0.045 for pulmonary management visits; and 0.37 vs 2.17, P = 0.004 for tracheostomy). This study provides the first real-world evidence of eteplirsen treatment effects on key health outcomes in DMD pts.