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Last updated on April 20, 2014 at 21:20 EDT

Latest Nonsense mutation Stories

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...

2013-11-21 08:27:31

Eloxx to focus on the discovery of small molecule therapeutics that selectively induces ribosomal read-through of premature termination codons for the treatment of nonsense genetic diseases HERZLIYA, Israel, November 21, 2013 /PRNewswire/ -- Eloxx Pharmaceuticals, Ltd. today announced the initial closing of its first round of financing with Roche (SIX: RO, ROG; OTCQX: RHHBY) and Pontifax, an Israeli VC firm leading the financing. Eloxx Pharmaceuticals is based on a...

2013-11-14 08:33:24

-- Conference call to be held today at 8:00 a.m. ET -- SOUTH PLAINFIELD, N.J., Nov. 14, 2013 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT), a biopharmaceutical company focused on the discovery and development of orally administered, proprietary small molecule drugs that target post-transcriptional control processes, today announced financial and corporate results for the quarter ended September 30, 2013. (Logo: http://photos.prnewswire.com/prnh/20010919/PTCLOGO ) "2013 has thus far...

2013-10-10 08:29:08

SOUTH PLAINFIELD, N.J., Oct. 10, 2013 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced that the following two posters will be presented at the 27th Annual North American Cystic Fibrosis Conference in Salt Lake City, Utah, on October 17-19: -- The use of chronic inhaled antibiotics in the phase 3 clinical trial of ataluren in patients with nonsense mutation cystic fibrosis (poster number 207) -- Use of different pulmonary exacerbation definitions in...

2013-09-25 16:26:33

SOUTH PLAINFIELD, N.J., Sept. 25, 2013 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced that four posters will be presented at the 18(th) International World Muscle Society Congress (WMS) in Asilomar, California, on October 1-5. The following posters will be presented on October 3 from 14:30 - 16:00 during Dystrophinopathy pharmco and pharmaco-gene therapy Session. -- Phase 2b, dose-ranging study of ataluren (PTC124®) in nonsense mutation Duchenne muscular...

2013-09-18 08:29:10

SOUTH PLAINFIELD, N.J., Sept. 18, 2013 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced that two abstracts have been accepted for the 10(th) European Pediatric Neurology Society Congress (EPNS) in Brussels, Belgium, on September 25-28. -- Design of a confirmatory phase 3, multicenter, randomized, double-blind, placebo-controlled study of ataluren in patients with nonsense mutation Duchenne muscular dystrophy; -- Disease-related symptoms and...

2013-08-26 08:25:20

-Two publications further validate the use of 6MWT as a primary endpoint in Duchenne Muscular Dystrophy (DMD) clinical trials- SOUTH PLAINFIELD, N.J., Aug. 26, 2013 /PRNewswire/ -- Data published in the medical journal Muscle & Nerve demonstrate the clinical meaningfulness of the 6-minute walk test (6MWT) as a primary endpoint to measure disease progression and walking ability in ambulatory Duchenne muscular dystrophy (DMD) trials. The research also showed that a range of 20 to...

2013-06-26 12:53:53

A drug developed to treat genetic diseases such as Duchenne muscular dystrophy and cystic fibrosis may need a radical rethink. In a new study published on 25 June in the open access journal PLOS Biology, researchers question the mechanistic basis of the drug called PTC124 (also known as Ataluren), casting doubt as to whether it has the molecular effects that are claimed for it. This may have implications for its effectiveness in treating genetic diseases. An estimated 10% of all human...

2013-03-07 12:27:23

-Funds to support Phase 3 confirmatory trials of ataluren- SOUTH PLAINFIELD, N.J., March 7, 2013 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) today announced the successful completion of a $60 million financing led by Brookside Capital Partners Fund, L.P. Joining Brookside as new investors in this financing are Adage Capital Management, Jennison Associates L.L.C. (on behalf of fund clients), Longwood Fund and additional top-tier institutional investors. In conjunction with the...

2012-12-06 08:27:06

SOUTH PLAINFIELD, N.J., Dec. 6, 2012 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) today announced that the European Medicines Agency (EMA) has validated a Marketing Authorization Application (MAA) seeking conditional approval for ataluren, an investigational new drug for the treatment of patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). Validation of the MAA confirms that the submission is complete and begins the EMA's Committee for Human Medicinal Products' (CHMP) review...