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PTC Therapeutics Announces Additional Study of Ataluren in Patients with Advanced Nonsense Mutation Duchenne/Becker Muscular Dystrophy
Date:1/19/2010

for assessing heart, lung and muscle function in patients with more advanced disease as we all continue to work to halt its progressive and debilitating effects on all affected individuals."

PTC announced in February 2009 that it has completed enrollment of a registration-directed clinical trial in patients with nonsense mutation Duchenne and Becker muscular dystrophy.  The trial is a multi-center, randomized, double-blind, placebo-controlled study to determine whether ataluren can improve walking, activity, muscle function, and strength and whether the drug can safely be given for a long period of time.  Results from this trial are expected to be available in the first half of 2010.

ABOUT DUCHENNE/ BECKER MUSCULAR DYSTROPHY

Duchenne and Becker muscular dystrophy (DBMD) are progressive, debilitating and life-threatening genetic disorders representing a single disease continuum with the same underlying cause.  Patients with DBMD do not produce adequate levels of dystrophin, an essential protein that is critical to the structural stability of skeletal and heart muscles.  DMD is the most common and most severe form of muscular dystrophy in children, and approximately 20,000 children worldwide are born with DMD annually (one of every 3,500 male children).  Patients with BMD typically experience initial symptoms later and have a slower disease progression.  Nonsense mutations cause about 10 to 15 percent of all DBMD cases which results in an estimated 1,900 boys and young men with nonsense mutation DBMD in the United States.  Further information regarding DBMD is available through the Muscular Dystrophy Association (

SOURCE PTC Therapeutics, Inc.
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