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Oct 08, 2012
 |  BC Week In Review  |  Clinical News  |  Clinical Results

Eteplirsen: Additional extension study data

Additional data from the open-label Study 4658-US-202 extension of the double-blind, U.S. Phase IIb Study 4658-US-201 trial in 12 patients aged 7-13 with DMD showed that both doses of eteplirsen for 48 weeks met the primary endpoint of increasing the percentage of dystrophin-positive muscle fibers from baseline. Specifically, once-weekly 30 mg/kg IV eteplirsen led to a 52.1% mean increase in dystrophin-positive fibers from baseline to week 48 (p<=0.001), while once-weekly 50 mg/kg IV eteplirsen led to a 41.7% mean increase from baseline to week 48 (p<=0.008). Furthermore, patients who received placebo for 24 weeks followed by low- or high-dose eteplirsen for an additional 24 weeks achieved a significant 38.3% mean increase in dystrophin-positive fibers from baseline (p<=0.009).

High-dose eteplirsen for 48 weeks also led to significantly better clinical outcomes as measured by a 6MWT at week 48 compared to placebo...

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