March 2015
Together we can Cure Duchenne!
PTC Announces Translarna™ Access Program In Duchenne Muscular Dystrophy for Siblings of Patients Participating in PTC Clinical Trials

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PTC Therapeutics, Inc. (NASDAQ: PTCT), announced on March 3 that it will make Translarna available for siblings of patients in certain open label PTC clinical trials for nonsense mutation Duchenne muscular dystrophy (nmDMD). Translarna will be made available to these siblings in advance of commercial availability in their regions, after a treating physician and the patient or guardian make a clinical decision to use Translarna, and consistent with any other applicable regulatory requirements.

"For families with more than one child suffering from nonsense mutation Duchenne muscular dystrophy, having only one child enrolled in our clinical trials with access to Translarna, can cause great distress for parents," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "Knowing this, we worked to find a solution to ensure those siblings who were not eligible to participate in the trials can still have similar access. We are eager to roll out this program and are committed to providing access to Translarna for all who may benefit."

Kate Bushby, Professor of Neuromuscular Genetics at Newcastle University said, "Though Duchenne muscular dystrophy is a rare disorder, the nature of an inheritable genetic disease means that there is an increased likelihood that siblings will be born with the same mutation. Translarna is the first treatment option available to treat the underlying cause of nonsense mutation DMD. It is exciting to see PTC's commitment to the patients contributing towards our understanding of the natural history of Duchenne and developing Translarna as a treatment for nmDMD."

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