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

ned to alleviate the symptoms of the disease. These treatments include chest physical therapy to clear the thick mucus from the lungs, antibiotics to treat lung infections, and a mucus-thinning drug designed to reduce the number of lung infections and improve lung function. In addition, the majority of cystic fibrosis patients take pancreatic enzyme supplements to assist with food absorption in digestion. There is a significant unmet medical need for treatments that address the underlying cause of CF. More information regarding CF is available through the Cystic Fibrosis Foundation (http://www.cff.org).

About PTC124

PTC124 is an orally delivered, investigational new drug discovered by PTC Therapeutics. The drug is being developed for the treatment of nonsense mutation genetic disorders. Nonsense mutations are single-point alterations in the genetic code that prematurely stop the mRNA translation process, leading to production of truncated, non-functional proteins. PTC124 induces the cellular translation machinery to read through nonsense mutations in mRNA, inducing production of full-length, functional proteins. PTC124 has demonstrated proof of concept in Phase 2a clinical trials. Across all clinical studies to date, PTC124 has been generally well tolerated. PTC124 is currently in Phase 2b development with the goal of demonstrating that increasing functional protein levels in patients with nonsense mutation genetic disorders will safely provide clinical benefits.

PTC124 has been granted orphan drug status by the FDA and the European Commission for the treatment of cystic fibrosis and Duchenne muscular dystrophy due to nonsense mutations. The FDA has also granted PTC124 Subpart E designation for expedited development, evaluation, and marketing.

PTC has an exclusive collaboration with Genzyme Corporation to develop and commercialize PTC124 outside the United States and Canada. The development o
'/>"/>

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

0

GOOD

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:6/19/2013)... 19, 2013 Pharmacyclics, Inc. (the "Company") (Nasdaq: ... New England Journal of Medicine ( NEJM ) ... the investigational oral Bruton,s tyrosine kinase (BTK) inhibitor ibrutinib ... small lymphocytic lymphoma (SLL). Ibrutinib was shown to be ... SLL, even in patients who were at high-risk due ...
(Date:6/19/2013)... , June 19, 2013 The Physician-Patient Alliance for ... fatigue. The need to urgently address this issue ... Sentinel Event Alert #50 , "Medical device alarm safety in ... between January 2008 and June 2012, there were 98 alarm ... in death (82%) , 13 resulted in permanent loss ...
(Date:6/19/2013)... 2013   Voices Against Brain Cancer (VABC), ... brain cancer, today announced that it has awarded Tocagen ... clinical investigation of Toca 511 & Toca FC in ... cancer (Grade 3 or Grade 4).  Tocagen is a ... cancer, with an initial focus on brain cancer. ...
Breaking Medicine Technology:Ibrutinib Study Results in Patients with Relapsed/Refractory Chronic Lymphocytic Leukemia Published in The New England Journal of Medicine 2Ibrutinib Study Results in Patients with Relapsed/Refractory Chronic Lymphocytic Leukemia Published in The New England Journal of Medicine 3Ibrutinib Study Results in Patients with Relapsed/Refractory Chronic Lymphocytic Leukemia Published in The New England Journal of Medicine 4Ibrutinib Study Results in Patients with Relapsed/Refractory Chronic Lymphocytic Leukemia Published in The New England Journal of Medicine 5Ibrutinib Study Results in Patients with Relapsed/Refractory Chronic Lymphocytic Leukemia Published in The New England Journal of Medicine 6Three Lessons Learned to Reduce Alarm Fatigue and Improve Alarm Management and Patient Safety in Hospitals 2Three Lessons Learned to Reduce Alarm Fatigue and Improve Alarm Management and Patient Safety in Hospitals 3Voices Against Brain Cancer Awards Grant to Tocagen to Support Investigation of Toca 511 & Toca FC in Patients with Recurrent Brain Cancer 2Voices Against Brain Cancer Awards Grant to Tocagen to Support Investigation of Toca 511 & Toca FC in Patients with Recurrent Brain Cancer 3
... LONG BRANCH, N.J., March 1, 2011 Pharma ChemOutsourcing today ... member companies of the USA,s various state biotechnology associations to ... would like to attend the September 12-15 show in New ... of hundreds of successful biotechnology companies over the past 12 ...
... Pharmaceuticals, Inc. (Nasdaq: OPTR ) today announced that ... Citi 2011 Global Health Care Conference on Thursday, March ... New York Hotel in New York City. Cowen and ... 2011 at 4:45 p.m. Eastern Time at The Boston Marriott ...
Cached Medicine Technology:Pharma ChemOutsourcing Targets BioNJ, PA Bio, MassBio, BayBio, SoCalBio, and NY Biotechnology Association Member Companies for September 2011 Show in New Jersey 2Optimer Pharmaceuticals to Present at March 2011 Investor Conferences 2
(Date:6/19/2013)... 2013 Chrysler made news recently when it ... 2.7 million Jeeps. The Jeep models in question are the ... according to the NHTSA are vulnerable to fire and explosion ... models are rear-mounted and have been alleged to catch fire ... Grand Cherokee with 32 rear-end crashes, which resulted in 44 ...
(Date:6/19/2013)... 19, 2013 Amber Roberson with ... Benefits, LLC, Native Care Health, LLC and Medcase, ... Health Underwriters’ (NAHU) new professional development course on ... This continuing education course for agents has been ... receive eleven education credits from the Missouri Department ...
(Date:6/19/2013)... New York, New York (PRWEB) June 19, 2013 ... involving pancreatitis and pancreatic cancer continue to mount in ... the U.S. Food & Drug Administration (FDA) recently held ... other diabetes drugs known as incretin mimetics. According ... may seek additional data from the makers of Januvia ...
(Date:6/19/2013)... Lawrence, KS (PRWEB) June 19, 2013 ... photographs is common with today’s computer software, it creates ... a form of misconduct. As an already published case ... of photo manipulation. , The Journal of Parasitology ... image manipulation in a 2004 article published in ...
(Date:6/19/2013)... by researchers at The University of Texas MD Anderson ... remarkable promise for the treatment of relapsed or refractory ... findings of the 18-center Phase 2 study were published ... . Previous interim findings were presented in December 2012 ... and Exposition. , Unprecedented results, fewer side ...
Breaking Medicine News(10 mins):Health News:Zinda & Davis PLLC: Why the Chrysler Recall Refusal Matters And May Set a Dangerous Precedent 2Health News:Zinda & Davis PLLC: Why the Chrysler Recall Refusal Matters And May Set a Dangerous Precedent 3Health News:RWI Benefits, LLC Becomes PPACA Certified 2Health News:Januvia Lawsuit News: Bernstein Liebhard LLP Comments on Recent FDA Meeting to Discuss Safety of Januvia and Other Incretin Mimetics 2Health News:Januvia Lawsuit News: Bernstein Liebhard LLP Comments on Recent FDA Meeting to Discuss Safety of Januvia and Other Incretin Mimetics 3Health News:Altered Photo in Journal Article Illustrates the Need for Manipulation Guidelines 2Health News:Ibrutinib continues strong showing against mantle cell lymphoma 2
... has increased its health budget by over 30 percent ... a new medical college//. ,"In a bid to ... medical education department budget to about Rs.1.52 billion. This ... billion," an official said. ,"The government not ...
... the June Journal of the American Society of Nephrology ... for chronic renal failure//. ,"Our results confirm an ... as an important causative factor in kidney disease," comments ... of the new study. ,Dr. Ejerblad and colleagues ...
... According to a new study by New York University School ... New York City are severely infected// with hepatitis B virus. ... the liver gets inflamed and can ultimately lead to cirrhosis ... 12 issue of Morbidity and Mortality Weekly, published by the ...
... new study, oral and intravenous antiviral drug may be ... ,Shingles, which is caused by varicella-zoster virus, is a ... at the University of Colorado and Health Sciences Center ... three women with moderate to severe pain following shingles. ...
... Middle school students have expressed a high degree of comfort ... schools are safer// , and make the students feel wanted. ... other schools. Psychologists from UCLA and UC ... than 70 sixth-grade classrooms in 11 Los Angeles public middle ...
... which is a severe form of food poisoning, now has ... team of researchers working at the University of Bristol//. ... researchers have narrowed down on a particular antibiotic called fosfomycin, ... effective in the laboratory, this drug has never been considered ...
Cached Medicine News:Health News:Kidney Failure three times pronounced with obesity 2Health News:Asian American Community Prone To Hepatitis B infection 2Health News:Asian American Community Prone To Hepatitis B infection 3Health News:Comfort Level, Maximum in Ethnically Diverse Schools 2Health News:Comfort Level, Maximum in Ethnically Diverse Schools 3
Indications For Usage: , Nasal surgery. , Ophthalmic surgery. , Facial surgery. , T&A surgery. ...
The Cuffed Endoflex endotracheal tube had a murphy eye, high volume, low pressure cuff., ,Endoflex endotracheal tubes are individual packaged in sterilized pouches, 10 units per box, 10 boxes pe...
... The new Level 1 H-1200 Fast Flow ... builds on the benefits of the existing Level ... Level 1 H-1200 Fast Flow Fluid Warmer incorporates ... clamping capability, while continuing to deliver warm fluids ...
Futuremeds Astotherm Plus Blood and Fluid Warmers meet American Blood Bank and ECRI standards., ,Astotherm is a durable warmer you can rely on for many years....
Medicine Products: