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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

. The other publication analyzes the baseline data from all DMD patients in the same Phase 2b study. The study comprised a 6-week screening period and a 48-week blinded study drug treatment period. Patients were randomized 1:1:1 to receive: ataluren at either high- or low-dose or matching placebo, daily for 48 weeks. Evaluations were performed at screening, baseline, and every 6 weeks for 48 weeks.

There were 174 randomized DMD patients included in the study. All patients screened and randomized were males, ranging in age from 5 to 20 years. Nonsense mutations were distributed across the span of the 79 exons of the dystrophin gene, with no mutational hotspots identified, and represented all three types of premature stop codons. Test-retest reliability was determined using the baseline and screening values for all endpoints. Concurrent validity and MCIDs were determined using pre-treatment data.

About Duchenne Muscular Dystrophy (DMD)
Primarily affecting males, Duchenne muscular dystrophy (DMD) is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of skeletal, diaphragm, and heart muscles. Patients with Duchenne muscular dystrophy, the more severe form of the disorder, lose the ability to walk as early as age 10 and experience life-threatening lung and heart complications in their late teens and twenties. There are an estimated 34,000 patients with DMD in the United States and Europe and approximately 13 percent of all DMD cases are caused by nonsense mutations in the dystrophin gene. More information about DMD is available through the Muscular Dystrophy Association (www.mdausa.org), Parent Project Muscular Dystrophy (www.parentprojectmd.org), Action Duchenne (www.actionduchenne.org), United
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SOURCE PTC Therapeutics, Inc.
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