Navigation Links
PTC Announces Data Showing That PTC124 Causes Statistically Significant Improvements in Chloride Channel Function in Cystic Fibrosis Patients
Date:10/24/2008

al trials that evaluate long-term clinical benefit for CF patients."

Patients with CF lack adequate levels of the CFTR protein, a chloride channel which is required for normal function of the lung, pancreas, liver and other organs. Nonsense mutations are single-point alterations in the genetic code that when transcribed into mRNA prematurely stop the mRNA translation process, preventing production of a full-length, functional protein. Patients with nonsense-mutation CF make virtually no CFTR protein and thus often have a more severe form of CF. Nonsense mutations are responsible for approximately 10 percent of the cases of CF worldwide.

By inducing the production of functional CFTR, PTC124 addresses one of the underlying genetic defects responsible for CF. Earlier studies of PTC124 in adults with CF evaluated nasal transepithelial potential difference (TEPD) as a surrogate for the presence and activity of the CFTR protein. Across a variety of studies, TEPD assessments showed statistically significant improvements of mean CFTR-dependent chloride secretion in the airways.

European Phase 2a Study Results

The multicenter, randomized, open-label Phase 2a study demonstrated that treatment with PTC124 caused improvements in CFTR-mediated chloride conductance and in apical expression of CFTR protein in nasal epithelium. The study enrolled 30 children ages 6 to 18 years to evaluate the activity, safety and pharmacokinetic activity of PTC124 in pediatric patients. All of the patients had nonsense-mutation CF, pathological lung infection, and CF-induced pancreatic insufficiency. In a cross-over design testing 14 days of PTC124 at two dose levels, patients were randomized to receive either the lower or higher dose first. PTC124 treatment across both dose levels was associated with a statistically significant (p=0.03) increase in the proportion of epithelial cells showing cell surface CFTR protein. PTC124 also induced significantly improved CFTR-media
'/>"/>

SOURCE PTC Therapeutics, Inc.
Copyright©2008 PR Newswire.
All rights reserved

Page: 1 2 3 4 5 6

Related medicine technology :

1. ULURU Inc. Announces the Commencement of First Clinical Study Using Altrazeal(TM) Silver
2. Raptor Pharmaceuticals Announces Phase 2a Clinical Trial Initiation in Non-Alcoholic Steatohepatitis (NASH)
3. BioMarin Announces Positive Results From Phase 2A Clinical Study of 6R-BH4 in Sickle Cell Disease
4. Sirion Therapeutics Announces Positive Pivotal Uveitis Data for Durezol(TM)
5. Arpida Announces Review of Iclaprim NDA by FDA Advisory Committee in November 2008
6. Neurobiological Technologies Announces FDA Agreement to Consolidate Viprinex Phase 3 Trials Accelerating Time to Pivotal Data
7. Facet Solutions Announces Completion of 6 Month Follow-Up in US Pilot Study
8. Quark Pharmaceuticals Announces Publication of Study on Use of siRNA against Proprietary Target for Inhibiting Tumor Growth and Sensitization of Cancer Cells to Chemotherapy
9. XTENT Announces Clinical Trial Data to be Presented at Annual Meeting of Transcatheter Cardiovascular Therapeutics Next Week
10. SCOLR Pharma Announces Completion of Patient Enrollment and Dosing for Pivotal Clinical Trial Evaluating Extended Release Ibuprofen
11. In celebration of World Hospice and Palliative Care Day, FHSSA Announces Seven New Partnerships Between U.S. Organizations and African Hospices
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:7/2/2015)... -- ResMed Inc. (NYSE: RMD ) today announced that ... June 30, 2015 results on Thursday, July 30, 2015, after ... with ResMed,s results will be issued after 1:00 p.m. US ... discuss operating results and future outlook. The earnings ... Time and the live webcast of the call can be ...
(Date:7/2/2015)... HILL, N.C. , July 2, 2015  New ... Because of its leading role in educating thought leaders ... functions that can heavily influence how well a new ... a new study from research and consulting leader Best ... Affairs leaders need to focus on are early stage ...
(Date:7/2/2015)... 2015  Fort Lauderdale-based investment banking firm, Farlie ... its Consumer Group completed the sale of Insight ... (ISE: KRZ) ("Kerry"), one of the leading global ... well as functional ingredients and actives for the ... served as the exclusive financial advisor to the ...
Breaking Medicine Technology:ResMed to Announce Fourth Quarter and Fiscal Year-End 2015 Results 2Medical Affairs Role in Thought Leader Education is Key Success Factor for New Product Launches in the Pharmaceutical Industry 2Farlie Turner's Consumer Group Completes Sale of Insight Beverages to Kerry 2
... 2011 /PRNewswire-Asia-FirstCall/ -- Tianyin Pharmaceutical Inc. (NYSE Amex: ... specializes in the development and sale of patented ... generics and active pharmaceutical ingredients (API) announced that ... Macrolide Facility (JCM) has passed the internal quality ...
... NEW YORK, Nov. 9, 2011 ... research report is available in its ... and Reimbursement in Europe - Reference ... to Innovative Drugs Pose Significant Challenge ...
Cached Medicine Technology:JCM Passes Internal Quality Assessment for Azithromycin API Production 2JCM Passes Internal Quality Assessment for Azithromycin API Production 3Pharmaceutical Pricing and Reimbursement in Europe - Reference Pricing and Similar Initiatives Being Applied to Innovative Drugs Pose Significant Challenge to Pharmaceutical Companies 2Pharmaceutical Pricing and Reimbursement in Europe - Reference Pricing and Similar Initiatives Being Applied to Innovative Drugs Pose Significant Challenge to Pharmaceutical Companies 3Pharmaceutical Pricing and Reimbursement in Europe - Reference Pricing and Similar Initiatives Being Applied to Innovative Drugs Pose Significant Challenge to Pharmaceutical Companies 4Pharmaceutical Pricing and Reimbursement in Europe - Reference Pricing and Similar Initiatives Being Applied to Innovative Drugs Pose Significant Challenge to Pharmaceutical Companies 5Pharmaceutical Pricing and Reimbursement in Europe - Reference Pricing and Similar Initiatives Being Applied to Innovative Drugs Pose Significant Challenge to Pharmaceutical Companies 6Pharmaceutical Pricing and Reimbursement in Europe - Reference Pricing and Similar Initiatives Being Applied to Innovative Drugs Pose Significant Challenge to Pharmaceutical Companies 7
(Date:7/2/2015)... (PRWEB) , ... July 02, 2015 , ... ... Donna Edwards (D-Md.) for her introduction of the Stop Overdose Stat (S.O.S.) Act ... by Senator Jack Reed (D-R.I.), this Act seeks to decrease drug overdose – ...
(Date:7/2/2015)... ... July 02, 2015 , ... ... retrofit project at the University of Minnesota, they knew it would save the ... also be an award-winning choice: the total energy savings after installation was an ...
(Date:7/2/2015)... RI (PRWEB) , ... July 02, 2015 , ... Many ... safety in mind. That’s why Amica Insurance is sharing some tips to ... Commission (CPSC), approximately 230 people go to the emergency room daily with fireworks-related injuries ...
(Date:7/2/2015)... ... ... With summer in full swing, Americans are flocking to Canada’s many lakes to reel in ... in the world, and it’s easy to understand why. From the Pacific Northwest Coast of ... Recreational fishing is a huge industry, and Americans make up the vast majority of anglers ...
(Date:7/2/2015)... ... July 02, 2015 , ... In 2012, Patrick Achebe realized that the gift of poetry was ... down to write his first poem, and three years later he was publishing an entire ... worth sharing with the world. "I have always believed that the best way to lift ...
Breaking Medicine News(10 mins):Health News:AAPCC Supports the Stop Overdose Stat Act of 2015 2Health News:AAPCC Supports the Stop Overdose Stat Act of 2015 3Health News:Kenall’s TekDek™ Helps University Achieve Award-Winning Energy Savings in Parking Structure Lighting Retrofit 2Health News:6 Fireworks Safety Tips from Amica Insurance 2Health News:Patrick Achebe Releases Debut Book, Whispering Waves: Poems for Everyday Grace 2
... performed, much has been learned about illegal transplants of kidneys ... quite common in countries like India and China where ... people sell their kidneys to foreigners. ,Much has ... was in Jan 2006, about an Indian who died in ...
... (PMA) plans to move court against the bans handed to ... ,"The Pakistan Cricket Board (PCB) has pleased the outside world ... but justice was not meted out to the two," PMA ... Monday., ,On Wednesday, a specially constituted PCB tribunal had ...
... hit harder by hangover while teenagers have a greater resistance ... Elena Varlinskaya and other researchers studied the effects of alcohol ... adults. , ,The rats - chosen because their brain ... and their movements monitored, reported the online edition of Daily ...
... on November 7 and the theme selected is Cancer ... a marathon on that day// to promote awareness and ... ,"Run 4 Cancer" will be flagged off ... Hospital. Commissioner Allahabad Division RN Tripathi will preside over ...
... visitor from the Safari country of Kenya. A 68 ... underwent bilateral// knee replacements here in a city hospital. ... this was the first time that a Kenyan citizen ... His son, a UK based accountant located the Doctor ...
... the organisation he earlier worked for has been arrested here, ... Police arrested Alok Tomar, a former editor of Senior India ... arrested after he flew into the capital Friday night. He ... Police Alok Kumar. ,Kumar said Senior India Pvt ...
Cached Medicine News:Health News:Older People Get Worse Hangovers 2Health News:Awareness about Women's Cancers- A National Concern 2
... Laboratories is a national reference laboratory and ... and development. ARUP offers an extensive test ... tests in clinical and anatomic pathology. Owned ... clients include more than half of the ...
... ARUP Laboratories is a national reference ... laboratory research and development. ARUP offers an ... unique medical tests in clinical and anatomic ... ARUP Laboratories' clients include more than half ...
... reference laboratory and a worldwide leader in ... an extensive test menu of highly complex ... anatomic pathology. Owned by the University of ... half of the nation's university teaching hospitals ...
... Laboratories is a national reference laboratory and ... and development. ARUP offers an extensive test ... tests in clinical and anatomic pathology. Owned ... clients include more than half of the ...
Medicine Products: