PTC Therapeutics Announces Data Showing Six-Minute Walk Test Consistently Measures Ambulatory Function in Patients With Duchenne Muscular...(cteristic of boys with DMDimpair their s...),Health

Date:9/30/2008

cteristic of boys with DMD impair their stride length and cadence, resulting in shorter 6-minute walk distances than healthy boys. The data correlate well with the known natural history of DMD and suggest that the test should be able to detect treatment effects on DMD-related muscular deficits.

"The 6MWT evaluates the critical human function of ambulation and is highly relevant to patients with DMD, who progressively lose walking ability during childhood," said Dr. McDonald. "Establishing a gold-standard outcome measure for new therapies in DMD has long been a goal among researchers. These findings allow us to confidently use changes in the distance achieved during the 6MWT as a major efficacy endpoint in therapeutic trials."

These findings directly support the design of an ongoing phase 2b, double-blind, randomized trial of PTC124 in boys with DMD/BMD, in which the 6MWT is the primary outcome measure. Demographic and baseline data from this study are also being presented at the WMS Congress today. This multicenter, international study is enrolling 165 ambulatory boys who are at least five years old. Today's presentation reports baseline data on the first 43 subjects, who range in age from 5 to 17 years. Using the 6MWT, subjects were tested and then retested up to six weeks later; the correlation between test and retest was high (r=0.93), further demonstrating the reliability of the 6MWT. In addition to the ongoing phase 2b study, PTC124 is being evaluated in an open-label, long-term extension study open to boys who participated in an earlier phase 2a trial.

"The findings from these clinical trials contribute provide strong underpinnings for the design of the PTC124 clinical trials program," said Langdon Miller, M.D., Chief Medical Officer of PTC Therapeutics, Inc. "We are hopeful that these data can benefit the entire field of DMD clinical research and therapeutics development and can establish regulatory precedent of use of the 6MWT in DMD/BMD
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SOURCE PTC Therapeutics, Inc. Copyright©2008 PR Newswire. All rights reserved

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