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Latest Nonsense mutation Stories

2014-07-09 16:28:13

-French ATU Cohort and Other Named Patient Programs Authorized - SOUTH PLAINFIELD, N.J., July 9, 2014 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the initiation of a reimbursed expanded access program (EAP). PTC's EAP program is intended to make Translarna(TM) (ataluren) available to patients before commercial availability in certain countries. Where mechanisms exist and in accordance with local regulations, PTC will make Translarna available to nonsense...

2014-06-30 08:30:32

SOUTH PLAINFIELD, N.J., June 30, 2014 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the initiation of a global confirmatory Phase 3 clinical trial of Translarna(TM) (ataluren), an investigational new drug, in patients with nonsense mutation cystic fibrosis (nmCF). Nonsense mutations within cystic fibrosis are categorized as Class I mutations, a severe form of CF that results in little or no production of the CFTR protein. The Phase 3 confirmatory trial is...

2014-06-30 08:30:29

SOUTH PLAINFIELD, N.J., June 30, 2014 /PRNewswire/ -- 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....

2014-05-23 08:23:53

- The first treatment for the underlying cause of Duchenne muscular dystrophy - SOUTH PLAINFIELD, N.J., May 23, 2014 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that following its request for re-examination, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion regarding the company's application for a conditional marketing authorization of Translarna(TM) (ataluren) for the treatment...

2014-05-16 08:26:28

- Data Demonstrate Positive Trends in Lung Function and Pulmonary Exacerbations - SOUTH PLAINFIELD, N.J., May 16, 2014 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the results of a Phase 3 study of ataluren in patients with nonsense mutation cystic fibrosis (nmCF) were published in Lancet Respiratory Medicine. The results demonstrated positive trends in both the primary endpoint, lung function as measured by relative change in % predicted FEV(1) (forced...

2014-01-24 08:23:47

SOUTH PLAINFIELD, N.J., Jan. 24, 2014 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) (PTC) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion on the Company's marketing authorization application (MAA) for conditional approval of ataluren for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). The response from the CHMP is consistent with the Company's previous...


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bibliopole
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