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PTC124 in Medical News

Drug fights cystic fibrosis

...A mutation. In the UAB tests performed on mice, ptc124 restored to normal function up to 29 percent of th...shed last year in the journal Nature, which showed ptc124 restored up to 25 percent of the missing or abnorm... Our study shows strong pre-clinical evidence that ptc124 is capable of suppressing nonsense mutations that ...

Scientists Explore ‘muscle’ in a New Drug to Assuage Muscular Dystrophy Symptos

...tal drug has raised scientists’ hope in being able to repair the genetic aberration, so typical among victims of Duchenne Muscular Dystrophy. The drug ptc124 proved its mettle in repairing the lost muscle function during experiments conducted on rodents. Human trial for this drug is underway, though it migh...

Potential Lifesaver Drug Announced

...rld is too thrilled for silence. The drug named as ptc124 and been worked upon by researchers from PTC Thera...ass of mutations.” So how does the drug work? ptc124 works by binding to a part of the cell called the ...being tried out on 100 humans. At the same time ptc124 is just one of a new generation of treatments whic...
PTC124 in Medical Technology

PTC Announces Data Showing That PTC124 Causes Statistically Significant Improvements in Chloride Channel Function in Cystic Fibrosis Patients

- PTC124 Highlighted in Multiple Presentations at North American Cystic Fibrosis Conference - SOUTH PLAINFIELD, N.J. and ORLANDO, Fla., Oct. 24 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) today announced that results from a randomized Phase 2a European study demonstrated that treatment with...

PTC Therapeutics Announces Data from Additional Clinical Studies of PTC124 in Cystic Fibrosis Confirming Activity

- Data Presented at the 31st European Cystic Fibrosis Conference - SOUTH PLAINFIELD, N.J. and PRAGUE, Czech Republic, June 12 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) today announced promising new data from two studies of PTC124 in cystic fibrosis (CF). Results from an Israeli Phase 2a ...

PTC Therapeutics Announces Initiation of Phase 2b Registration-Directed Clinical Trial of PTC124 in Duchenne/Becker Muscular Dystrophy

First Registration Study of an Investigational Drug for Duchenne/Becker Muscular Dystrophy SOUTH PLAINFIELD, N.J., April 23 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC), today announced the initiation of an international pivotal trial of PTC124 in patients with Duchen...

PTC Therapeutics Announces Additional Results from Phase 2 Study of PTC124 in Duchenne Muscular Dystrophy

Data Presented at the World Muscle Society International Congress SOUTH PLAINFIELD, N.J., Oct. 18 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery, development, and commercialization of small-molecule drugs targeting post-transcriptional con...

PTC Therapeutics Announces Pharmacokinetic and Safety Results from Phase 2 Study of PTC124 in Duchenne Muscular Dystrophy

- Data Presented at 36th Child Neurology Society Annual Meeting - SOUTH PLAINFIELD, N.J., Oct. 11 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery, development and commercialization of small-molecule drugs targeting post-transcriptional cont...

PTC Therapeutics Announces $25 Million Award From Cystic Fibrosis Foundation Therapeutics for Development of PTC124

- Expanded Collaboration Supports Pivotal Phase 2b Clinical Trials of First Potential Therapy to Address an Underlying Cause of Cystic Fibrosis - SOUTH PLAINFIELD, N.J. and BETHESDA, Md., July 16 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) and Cystic Fibrosis Foundation Therapeutics, Inc. (C...

PTC Therapeutics Announces Additional Positive Interim Phase 2 Results of PTC124 in Duchenne Muscular Dystrophy

Data Presented at 59th American Academy of Neurology Annual Meeting BOSTON and SOUTH PLAINFIELD, N.J., May 04, 2007 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery and development of small-molecule drugs targeting post-transcriptional control proc...

PTC Therapeutics Completes Enrollment of Pivotal Trial in Muscular Dystrophy

... - Ataluren designated as generic name for ptc124 - SOUTH PLAINFIELD, N.J., Feb. 3 /PRNewswire/...atient enrollment of its pivotal clinical trial of ptc124 in patients with nonsense mutation Duchenne and Be...f schedule. The company also announced today that ptc124 has been issued the generic name, ataluren. (...

PTC Therapeutics Announces Data Showing Six-Minute Walk Test Consistently Measures Ambulatory Function in Patients With Duchenne Muscular Dystrophy

...ngoing phase 2b, double-blind, randomized trial of ptc124 in boys with DMD/BMD, in which the 6MWT is the pri...e 6MWT. In addition to the ongoing phase 2b study, ptc124 is being evaluated in an open-label, long-term ext...provide strong underpinnings for the design of the ptc124 clinical trials program," said Langdon Miller, M.D...

PTC Therapeutics Announces Data Supporting Cough Frequency as a New Outcome Measure in Evaluating Treatments for Cystic Fibrosis

...e look forward to presenting data on the effect of ptc124 on cough frequency at the 31st annual European Cys... year to further evaluate the clinical efficacy of ptc124 in patients with CF." About Cystic Fibrosis Cy...oundation (http://www.cff.org ). About PTC124 ptc124 is an orally delivered investigational new drug in...
PTC124 in Biological News

PTC124 shows activity in cystic fibrosis; Phase 2 proof-of-concept data published in Lancet

...he Lancet show that the investigational oral drug ptc124 demonstrates activity in nonsense-mutation cystic fibrosis (CF). The data show that treatment with ptc124 results in statistically significant improvements ...F. By inducing the production of functional CFTR, ptc124 is addressing the underlying genetic defect respon...
PTC124 in Biological Technology

PTC Therapeutics Announces Encouraging Additional Phase 2 Results of PTC124 in Cystic Fibrosis

- New Pediatric Data Presented at 21st North American Cystic Fibrosis Conference Confirm Previous Findings in Adult population - SOUTH PLAINFIELD, N.J., Oct. 5 /PRNewswire / -- PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery, development and commercial...

PTC Therapeutics Receives $2.6 Million in Government Grants

...al studies to date. The FDA and the European Commission have granted ptc124 (ataluren) orphan drug status for the treatment of DMD and cystic fibrosis due to nonsense mutations. The FDA has also granted ptc124 (ataluren) Subpart E designation for expedited development, evaluation, and...

PTC124 Shows Promising Activity in Cystic Fibrosis; Phase 2 Proof-of-Concept Data Published in the Lancet

...c fibrosis (CF). The data show that treatment with ptc124 results in statistically significant improvements ...CF. By inducing the production of functional CFTR, ptc124 is addressing the underlying genetic defect respon...ble for the majority of CF cases. Study results: ptc124 Restored Functional Production of CFTR Protein in ...

Genzyme Corporation and PTC Therapeutics Announce Collaboration on Small Molecule for Genetic Diseases

... - Potential New Treatment Paradigm, ptc124 - CAMBRIDGE, Mass. and SOUTH PLAINFIELD, N.J., J...the terms of the agreement, PTC will commercialize ptc124 in the United States and Canada, and Genzyme will ...ally applicable to hundreds of genetic diseases. ptc124 is currently being evaluated in a phase 2b trial f...

PTC124 Featured at Third Annual Congress of Myology

.../www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO ) ptc124 is being studied in Duchenne muscular dystrophy (D...scular dystrophy (BMD) to evaluate the efficacy of ptc124 as measured by improvements in the walking ability...e of this disease," stated Dr. Voit. "The Phase 2b ptc124 clinical trial sets a gold standard for future cli...
PTC124 in Biological Dictionary

Nonsense Mutation

...on . deletion. all of the above. none of the above ... Nonsense mutations are a common cause of human genetic disease. ... ER, Zhuo J, et al. ptc124 targets genetic disorders caused by nonsense mutations . ... ...
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