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New Evidence Published in Muscle and Nerve on Measuring 6-Minute Walk Distance in Duchenne Muscular Dystrophy
Date:8/26/2013

ient population that are in the decline phase of walking ability and would best demonstrate benefit in a 48-week trial. When we looked back at the subgroup of 61 patients from our completed Phase 2b trial that fit the inclusion criteria of the current trial, they showed an average 50 meter improvement in ambulation as measured in the 6-minute walk distance from patients on placebo. We anticipate that the Phase 3 trial will complete enrollment mid-2014."

This report in Muscle & Nerve represents an evaluation of the largest multicenter dataset collected to date to determine the reliability and concurrent validity of the 6MWT and other clinical endpoints in DMD. In addition, the research assesses the distribution-based MCID (minimal clinically important difference) for 6MWD and other commonly-employed functional endpoints (eg. timed function tests and quantitative knee extension strength measures) as well as the 6MWD, which is the most common primary endpoint for ambulatory DMD clinical trials. Moreover, baseline walking ability, as measured by the 6MWD was found to be predictive of time to ≥10% worsening in function, a clinically meaningful milestone, in DMD.

The research findings confirm that the 6MWT is a safe and feasible measure of walking ability in DMD and a valid primary outcome measure for ambulatory DMD clinical trials. In addition, the data supports the clinically meaningful change in walking ability, as measured by 6MWT, to be in the range of 20 to 30 meters, which can serve as a targeted treatment effect in 12-month trials in ambulatory DMD.

About the Publications           
The natural history data were derived from the placebo arm of a Phase 2b, international, multicenter, randomized, double-blind, placebo-controlled, dose-ranging study to evaluate the efficacy and safety of ataluren in ambulatory male patients with nonsense mutation DMD aged ≥5 years
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SOURCE PTC Therapeutics, Inc.
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