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Latest Nonsense mutation Stories

2014-09-17 08:29:28

SOUTH PLAINFIELD, N.J., Sept. 17, 2014 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the expansion of its global presence with the addition of key executives to leadership positions in PTC's global commercial team and the establishment of its international headquarters in Dublin, Ireland. Significant pre-launch activities are well underway as PTC prepares for the commercial launch of Translarna(TM) (ataluren) in the European Union. In August of this year,...

2014-09-10 12:28:50

However, Less Than One-Third of Duchenne Patients Are in Subpopulations That Will Benefit From New Disease-Modifying Therapies, According to Findings from Decision Resources Group BURLINGTON, Mass., Sept. 10, 2014 /PRNewswire/ -- Decision Resources Group finds that the first three emerging disease-modifying therapies (DMTs)--PTC Therapeutics' Translarna (ataluren), Prosensa's drisapersen and Sarepta's eteplirsen--will likely receive regulatory approval for Duchenne muscular dystrophy (DMD) in...

2014-09-09 08:31:34

-Top-line data expected H2 2015- SOUTH PLAINFIELD, N.J., Sept. 9, 2014 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that it has completed enrollment of ACT DMD, the Phase 3 confirmatory trial of Translarna(TM) (ataluren) for patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). Top-line data from the trial is expected in the second half of 2015 and will support further approvals globally, following European approval received earlier this...

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...


Word of the Day
humgruffin
  • A terrible or repulsive person.
Regarding the etymology of 'humgruffin,' the OED says (rather unhelpfully) that it's a 'made-up word.' We might guess that 'hum' comes from 'humbug' or possibly 'hum' meaning 'a disagreeable smell,' while 'gruffin' could be a combination of 'gruff' and 'griffin.'