Navigation Links
Top-line Data From Phase 3 Trial of Ataluren in Patients with Nonsense Mutation Cystic Fibrosis Show Promising Results
Date:6/8/2012

SOUTH PLAINFIELD, N.J., June 8, 2012 /PRNewswire/ -- PTC Therapeutics, Inc. today announced the results from a Phase 3 study of ataluren, an investigational new drug, in patients with nonsense mutation cystic fibrosis (nmCF). The results demonstrated positive trends in lung function as measured by FEV1 (forced expiratory volume in one second) and in the secondary outcome measure, rate of pulmonary exacerbations. Results also showed a substantial ataluren treatment effect in FEV1 and exacerbation rate in the pre-specified subpopulation of patients not receiving chronic inhaled antibiotics, representing approximately one half of the study population. These data were presented at the European Cystic Fibrosis Society Conference in Dublin, Ireland.

(Logo: http://photos.prnewswire.com/prnh/20010919/PTCLOGO )

"The data from this trial are promising. Ataluren has the potential to provide benefit to patients with nonsense mutation cystic fibrosis, who currently have few treatment options. Nonsense mutations do not produce any functional CFTR protein and therefore generally result in a more severe form of cystic fibrosis," stated Michael Konstan, M.D., a principal investigator at University Hospitals Rainbow Babies and Children's Hospital in Cleveland, Ohio. "Correcting a nonsense mutation in the cystic fibrosis transmembrane conductance regulator gene is a big challenge. Over 48 weeks, the decrease in FEV1 in ataluren was 50% less than the decrease on placebo, which is a substantial difference. It is also very important to note that the FEV1 and exacerbations tracked closely." 

This Phase 3 study, which was conducted across 11 countries, was a double-blind, placebo-controlled study comparing ataluren (n=116) to placebo (n=116) in nmCF patients. The primary endpoint, the relative change from baseline in %-predicted FEV1 at 48 weeks, showed a positive trend favoring atalur
'/>"/>

SOURCE PTC Therapeutics, Inc.
Copyright©2012 PR Newswire.
All rights reserved

Page: 1 2 3 4 5 6

Related medicine technology :

1. Amgen Announces Top-Line Results Of Phase 3 Sensipar®/Mimpara® EVOLVE™ Trial
2. Omthera Pharmaceuticals Announces Positive Top-Line Results from Phase 3 EVOLVE Study
3. Lilly and Incytes Oral JAK1 and JAK2 Inhibitor, Baricitinib, Showed Positive Results in Phase IIb Study in Patients with Active Rheumatoid Arthritis
4. Sorrento Therapeutics Awarded Third Phase I STTR Grant
5. Taiho Pharmaceutical Announces Start of Global Phase III Clinical Trial in Metastatic Colorectal Cancer with Novel Antitumor Agent TAS-102
6. Interim Results Reported for Phase I Open-Label Clinical Trial for the Treatment of PTSD and MDD Using External Trigeminal Nerve Stimulation (eTNS™) - The USB Port to the Brain™
7. Flexions Lead Osteoarthritis Drug Candidate Demonstrates Significant, Prolonged Improvement in Pain and Function in Phase 2 Trial
8. Marshall Edwards Announces First Cohort Of Patients Dosed In Phase I Clinical Trial Of Oncology Drug Candidate ME-344
9. Preliminary Phase 2 Data of Ocera Therapeutics OCR-002 for the Treatment of Liver Cirrhosis and Upper GI Bleeding Show Potent Ammonia Reduction
10. Oncos Therapeutics Initiates Phase I Clinical Study of CGTG-102, an Oncolytic Adenovirus for the Treatment of Solid Tumor Cancers
11. Clinilabs Opens Phase I Unit In Eatontown, New Jersey
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:7/24/2014)...  Uroplasty, Inc. (NASDAQ: UPI ), a ... innovative proprietary products to treat voiding dysfunctions, today ... quarter ended June 30, 2014.  Global revenue ... grew 19% to $4.1 million, as compared to ... prior year.  Total revenue for the fiscal first ...
(Date:7/24/2014)... VIEW, Calif. , July 24, 2014 /PRNewswire/ ... a leading provider of medication and supply management ... a conference call Thursday, July 31, to discuss ... , Omnicell Second Quarter 2014 earnings conference ... 1:30 p.m. PTWho:  , Randall Lipps, chairman, president ...
(Date:7/24/2014)... 2014  Alimera Sciences, Inc. (NASDAQ: ALIM ... research, development and commercialization of prescription ophthalmic pharmaceuticals, today ... fiscal year 2014 financial results after the market close ... will follow on the same day at 4:30 p.m. ... hosted by Dan Myers , President and Chief ...
Breaking Medicine Technology:Uroplasty Reports Fiscal First Quarter Results 2Uroplasty Reports Fiscal First Quarter Results 3Uroplasty Reports Fiscal First Quarter Results 4Uroplasty Reports Fiscal First Quarter Results 5Uroplasty Reports Fiscal First Quarter Results 6Uroplasty Reports Fiscal First Quarter Results 7Uroplasty Reports Fiscal First Quarter Results 8Uroplasty Reports Fiscal First Quarter Results 9Uroplasty Reports Fiscal First Quarter Results 10Uroplasty Reports Fiscal First Quarter Results 11Omnicell to Release Second Quarter 2014 Earnings Results on July 31 2Alimera Sciences To Release Second Quarter 2014 Results 2
... WINDSOR, N.J. , July 7 ITelagen, a leading ... it was selected by Zweena, LLC of West Windsor, NJ ... ITelagen, which was selected for its expertise in EHR management, is ... solution. , , , ...
... DENVER , July 7 Invision Sally Jobe ... a new breast ultrasound technology for the early detection of cancer. Screening ... in women with radiologically dense breasts and other risk factors. , ... "Screening breast ultrasound is part ...
Cached Medicine Technology:Zweena Selects ITelagen to Host Personal Health Records Application 2Zweena Selects ITelagen to Host Personal Health Records Application 3Invision Sally Jobe Introduces New Breast Cancer Screening Technology to Denver 2Invision Sally Jobe Introduces New Breast Cancer Screening Technology to Denver 3Invision Sally Jobe Introduces New Breast Cancer Screening Technology to Denver 4
(Date:7/24/2014)... MEMPHIS, Tenn. Researchers at Le Bonheur Children,s Hospital ... results of a clinical trial of a new drug ... illness of healthy adult volunteers intranasally infected with respiratory ... of lower respiratory tract infections in young children in ... comes from the labs of Infectious Disease Specialist John ...
(Date:7/24/2014)... 24, 2014 In signing the ... Obama cited the Atlanta BeltLine Workforce Partnership in ... across the country ( http://www.whitehouse.gov/ready-to-work ). The ... pillar of U.S. economic strategy and profiled successful ... recognition from President Obama highlights the power of ...
(Date:7/24/2014)... with high-throughput data generated by breast cancer biologists ... computational method to determine how gene networks are ... as they respond to potential cancer therapy agents., ... each other in laboratory-grown cells is described in ... PLOS Computational Biology . , The method ...
(Date:7/24/2014)... rare procedure occasionally performed during Jewish circumcisions that involves ... herpes simplex virus type 1 (HSV-1) transmissions documented in ... conducted by Penn Medicine researchers and published online ... Society found. The reviewers, from Penn,s ... in New York, Canada and Israel. , The practiceknown ...
(Date:7/24/2014)... E. Taylor, M.D., director of The Miriam Hospital,s ... 2014 Rhode Island Medical Journal special ... c virus infection (hep c or HCV) is ... address other health challenges, and improve health care ... States, Taylor says, include lack of funding earmarked ...
Breaking Medicine News(10 mins):Health News:RSV research breakthrough to help infected children 2Health News:President Cites Atlanta BeltLine Workforce Partnership in Healthcare as National Model 2Health News:President Cites Atlanta BeltLine Workforce Partnership in Healthcare as National Model 3Health News:President Cites Atlanta BeltLine Workforce Partnership in Healthcare as National Model 4Health News:President Cites Atlanta BeltLine Workforce Partnership in Healthcare as National Model 5Health News:Gene changes in breast cancer cells pinpointed with new computational method 2Health News:Gene changes in breast cancer cells pinpointed with new computational method 3Health News:Link between ritual circumcision procedure and herpes infection in infants examined 2Health News:Miriam Hospital physician advocates awareness, collaboration to combat peaking hep C virus 2Health News:Miriam Hospital physician advocates awareness, collaboration to combat peaking hep C virus 3Health News:Miriam Hospital physician advocates awareness, collaboration to combat peaking hep C virus 4
... Children who can read and have good phonetic skills - ... may still be poor spellers. In a paper published in ... Annett, at the School of Psychology of Leicester, UK, show ... be right-handed than other poor spellers. , The three-year study ...
... May 29 Alsius Corporation,(Nasdaq: ALUS ... (IVTM(TM)) therapies for critically ill patients, was ... L.P. to provide,intravascular temperature management products to ... the fifth contract we have been awarded ...
... to what is known about relationship between fitness and,diabetes, ... 2 diabetes in women is significantly increased as a ... Index (BMI), and a,combination of the two increases the ... at the 55th Annual Meeting of the American,College of ...
... a first for URAC, the,independent, non-profit accreditation organization ... has developed to help case management,practitioners assess patient ... instrument is a standardized assessment case managers,can voluntarily ... from any interested,parties, including purchasers, policy makers, consumers, ...
... that they have coordinated all the entertainment for Rhinestone ... Hotel Indigo, on,West End, from 7pm until midnight., ... a,place for you to connect with artists, friends and ... the world of country music quick, easy,and free!, ...
... firefighters study to be presented at ... American College of Sports Medicine, MISSOULA, ... Exercise Metabolism (Montana WPEM) will present a human,clinical study at ... that wildland firefighters who consumed a yeast based,(beta glucan) antioxidant ...
Cached Medicine News:Health News:Alsius Corporation Awarded Three-Year Agreement With Premier, Inc. 2Health News:Alsius Corporation Awarded Three-Year Agreement With Premier, Inc. 3Health News:Studies Define Type 2 Diabetes Risk Factors in Women, Adolescents 2Health News:Studies Define Type 2 Diabetes Risk Factors in Women, Adolescents 3Health News:Studies Define Type 2 Diabetes Risk Factors in Women, Adolescents 4Health News:URAC Develops New Case Management Patient Satisfaction Tool, Invites Public Input On Voluntary Survey Instrument 2Health News:URAC Develops New Case Management Patient Satisfaction Tool, Invites Public Input On Voluntary Survey Instrument 3Health News:DigitalRodeo.com Lines Up Entertainment for Rhinestone Cure 2Health News:Montana Center for Work Physiology and Exercise Metabolism Presents Firefighter Health Study 2
Intended for the quantitative determination of direct bilirubin in serum. Modified EvelynMalloy Method. Endpoint Reaction. Wavelength: 555 nm. Linearity: 20 mg/dl (340 mol/l)...
... Powerwave is the first microplate spectrophotometer ... day, 7 days per week. With rugged ... footprint. Powerwave breaks the barrier of traditional ... reliably. One instrument can easily read up ...
Spectra 3000RS & Spectra 3000RSP are very precise instruments. They have a metal base structure, durable parts for long life use, and they are very easy to use and maintain....
... Series Spectrophotometer is truly ... UV-VIS instrumentation. The latest ... into a radically new ... and performance unprecedented in ...
Medicine Products: