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PTC Therapeutics Gets Parent Project Muscular Dystrophy's 'Path to Progress' Award

SOUTH PLAINFIELD, NJ – June 30, 2014 – PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that it is the recipient of Parent Project Muscular Dystrophy's (PPMD) "Path to Progress" award, honoring PTC's 16 years of commitment to bring the first-ever therapy for the underlying cause of the disorder to patients with Duchenne muscular dystrophy (DMD). The award was presented at PPMD's Annual Connect Conference in Chicago, Illinois.

"We are honored to be recognized by an organization that has had a tremendous impact on advancing the research and development of treatments for DMD," stated Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "PTC is committed to bringing new treatments to patients that are suffering from rare and neglected diseases. Early in PTC's history we became involved in pursuing new treatments for Duchenne patients and worked with PPMD to understand how we can best serve the community. We are extremely proud to be bringing Translarna to patients and grateful to the patients, families, advocacy groups, scientists and physicians who have supported us, and continue to do so, through the many years of research and development of Translarna. The CHMPs positive opinion is very gratifying, and we are committed to working towards providing access to all DMD patients."

In May 2014, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency adopted a positive opinion regarding PTC's application for a conditional marketing authorization of TranslarnaTM (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy. This was the first-ever recommendation by a regulatory body for approval of a therapy targeting Duchenne muscular dystrophy. Translarna (ataluren) is an investigational new drug discovered and developed by PTC.

"We congratulate PTC Therapeutics on its dedication to the Duchenne community and its research and development for compounds for this devastating disease," stated Pat Furlong, President & CEO of PPMD. "We applaud PTC for its perseverance in pursuing regulatory options to provide Translarna to patients as rapidly as possible. The company's pioneering work has paved the way and encouraged the scientific community to develop new therapies for DMD."

ABOUT PTC THERAPEUTICS, INC. PTC is a biopharmaceutical company focused on the discovery and development of orally administered, proprietary small molecule drugs that target post-transcriptional control processes. Post-transcriptional control processes regulate the rate and timing of protein production and are essential to proper cellular function. PTC's internally discovered pipeline addresses multiple therapeutic areas, including rare disorders, oncology and infectious diseases. PTC has developed proprietary technologies that it applies in its drug discovery activities and in collaborations with leading biopharmaceutical companies. For more information on the company, please visit our website


FORWARD LOOKING STATEMENTS: This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements, other than those of historical fact, contained in this release, are forward-looking statements including statements concerning the future expectations, plans and prospects for PTC Therapeutics, Inc. (PTC); the potential advantages of Translarna (ataluren); the timing of regulatory approvals, including any determination (whether positive or negative) by the European Commission (EC) with respect to conditional marketing authorization for Translarna in nonsense mutation Duchenne muscular dystrophy (nmDMD); and the objectives of management. Other forward-looking statements may be identified by the words "anticipate," "believe," "look forward," "expect," "intend," "may," "plan," "potential," "will," "would," "could," "should," "continue," and similar expressions. Our actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements we make as a result of a variety of risks and uncertainties, including, among others, those related to the initiation and conduct of clinical trials; availability of data from clinical trials; expectations for regulatory approvals (including the EC's determination with respect to conditional marketing authorization for Translarna in nmDMD); our scientific approach and general development progress; the availability or commercial potential of our product candidates; and other factors discussed in the "Risk Factors" section of PTC's most recent Form 10-Q and in PTC's other filings with the SEC. You are urged to carefully consider all such factors. The forward-looking statements contained herein represent PTC's views only as of the date of this press release, and we do not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this release.


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