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PTC Therapeutics Announces European Medicines Agency Validation of Marketing Authorization Application for Ataluren in Duchenne Muscular Dystrophy
Date:12/6/2012

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PTC also is currently conducting ongoing open-label safety and tolerability studies of ataluren for patients who were in previous ataluren clinical trials for nmDMD in both the United States and Europe. The company has worldwide development and commercial rights for ataluren in all indications and territories. Ataluren is also being developed for nonsense mutation cystic fibrosis and PTC is in discussions with regulators regarding next steps.

About Conditional Approval
Conditional approval is granted based on a positive benefit/risk ratio in the available data which, while not yet comprehensive, indicate that the public health benefits of immediate availability of a medicine outweigh its risks. The company is given obligations to fulfill, such as the performance of further studies. The approval is renewed on a yearly basis until all obligations have been fulfilled, and is then converted from a conditional approval into a full approval. Conditional approvals can only be granted for medicines that satisfy an unmet medical need, meaning the medicine is intended to be used for a disease or condition for which no treatment is readily available, and it is therefore important that patients have early access to the medicine concerned.

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 35,000 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 th
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SOURCE PTC Therapeutics, Inc.
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