Navigation Links
Ataluren Phase 2 Data in Nonsense Mutation Cystic Fibrosis Published in the European Respiratory Journal
Date:7/1/2011

rely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as the CFTR protein in nonsense mutation cystic fibrosis.

The development of ataluren has been supported by grants from Cystic Fibrosis Foundation Therapeutics, Inc. (the nonprofit affiliate of the Cystic Fibrosis Foundation); FDA's Office of Orphan Products Development; Muscular Dystrophy Association; National Center for Research Resources; National Heart, Lung, and Blood Institute; and Parent Project Muscular Dystrophy. The FDA and the European Commission have granted ataluren Orphan Drug status for the treatment of nonsense mutation cystic fibrosis and nonsense mutation Duchenne and Becker muscular dystrophy. The FDA has also granted ataluren Subpart E designation for expedited development, evaluation, and marketing for CF and dystrophinopathy and Fast Track designation for the development of treatment for nonsense mutation dystrophinopathy.

COLLABORATION WITH GENZYME

PTC Therapeutics has an exclusive collaboration with Genzyme Corporation for the development and commercialization of ataluren. PTC Therapeutics will commercialize ataluren in the United States and Canada, while Genzyme will commercialize the product in other regions of the world.

ABOUT CYSTIC FIBROSIS (CF)

CF is a life-threatening genetic disorder that causes serious lung infections and digestive complications. The predicted median age of survival for a person with CF is about 37 years.  According to the Cystic Fibrosis Foundation, CF affects approximately 30,000 adults and children in the United States and nearly 70,000 people worldwide. Genetic testing is required to confirm a complete diagnosis and to determine if a patient's disease is caused by a nonsense mutation. It is estimated that nonsense mutations are the cause of CF in about 10 percent of patients in t
'/>"/>

SOURCE PTC Therapeutics, Inc.
Copyright©2010 PR Newswire.
All rights reserved

Page: 1 2 3 4 5 6

Related medicine technology :

1. PTC Therapeutics Completes Enrollment of Phase 3 Trial of Ataluren in Patients with Cystic Fibrosis
2. PTC Therapeutics Announces Additional Study of Ataluren in Patients with Advanced Nonsense Mutation Duchenne/Becker Muscular Dystrophy
3. Endo Announces Topline Results From Phase 2 Study of Axomadol in Chronic Low Back Pain
4. Phase III Study Shows Once-Daily NVA237 is Superior to Placebo and Similar to Tiotropium in Improving Lung Function in COPD
5. DNA Medicine Institute Awarded Two Phase III SBIR NASA Contracts for Its rHEALTH Sensor
6. Phase I of the Swedish/Issaquah Medical Center Campus to Open in July
7. The Lancet Publishes Data from Protege, MacroGenics Phase 3 Clinical Study of Teplizumab in Type 1 Diabetes Patients
8. Morphotek®, Inc. Announces Initiation of Farletuzumab Phase II Study in First-Line Treatment of Non-Small Cell Lung Cancer
9. Palatin Technologies, Inc. Initiates Enrollment in Phase 2b Trial With Bremelanotide in Women With Female Sexual Dysfunction
10. Cardium Announces Plans to Acquire Transdel Pharmaceuticals Phase 3 Topical Analgesic and Cosmeceutical Business Assets
11. Glycotex, Inc. Releases Final Phase II Clinical Trials Results in the Clinicaltrials.gov Database and Announces the Results of the Mechanism of Action Study for its Product Candidate GLYC-101
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:11/21/2014)... , Nov. 21, 2014  The University of ... host the ceremonial groundbreaking of the new A. James ... The new building will cultivate transformative new engineering and ... Taking place at 10:00 a.m. on November 21 ... Paint Branch Parking Lot, adjacent to the Jeong H. ...
(Date:11/21/2014)... Calif. , Nov. 21, 2014 In ... and Performance Scrubs carried out a new product development ... industry,s very first "limited edition" lab coat , ... female professors of surgery in the world. The new ... its unisex predecessors, with a sleek envelope collar, vertical ...
(Date:11/21/2014)... Ill. , Nov. 21, 2014  Heated dialogue ... drug prices – from cancer treatments to Gilead,s new ... pharmaceutical leaders around the world questioning what it means ... of care. And while drug pricing ... pharmaceutical industry – from both a general business and ...
Breaking Medicine Technology:University of Maryland Breaks Ground for A. James Clark Hall 2University of Maryland Breaks Ground for A. James Clark Hall 3University of Maryland Breaks Ground for A. James Clark Hall 4Medelita Introduces Medical Industry's First Limited Edition Women's Designer Lab Coat 2"The Price of Global Health" Tackles Worldwide Drug-Pricing Debate 2"The Price of Global Health" Tackles Worldwide Drug-Pricing Debate 3"The Price of Global Health" Tackles Worldwide Drug-Pricing Debate 4"The Price of Global Health" Tackles Worldwide Drug-Pricing Debate 5
... Systems, Inc. (Nasdaq: DCTH ) announced that it ... recent corporate developments and update its progress on Monday, November ... Chief Executive Officer will host the call. ... domestic participants and 617-614-4927 for international participants, both using passcode ...
... 28, 2011 Mentor Foundation USA , the leading ... National Institute on Drug Abuse and The Embassy of Sweden ... together top scientists, parents, educators and community members to address ... The seminar will be held on Wednesday, Nov. 2 ...
Cached Medicine Technology:Delcath Systems to Report Third Quarter Progress and Conduct Conference Call 2Delcath Systems to Report Third Quarter Progress and Conduct Conference Call 3Escalating Trend of Prescription Drug Abuse Among Youth Seminar Hosted by Mentor Foundation USA 2
(Date:11/24/2014)... Dallas, TX (PRWEB) November 24, 2014 ... ADP ribose polymerase (PARP) and is one of the ... genetically vulnerable to creating some types of melanoma, and ... but this also sometimes gives their malignancies a exclusive ... on PARP to fix their DNA and allow them ...
(Date:11/24/2014)... 2014 The typical American diet includes ... ingredients. Consistently consuming a diet high in calories and ... excess fat that typically accumulate around the internal organs ... ugly head in the form of love handles, beer ... the decision to rid themselves of the burden of ...
(Date:11/24/2014)... "My friend was killed when a ... the street in his motorized wheelchair," said an inventor ... came up with this idea so that people in ... developed the Safety Lites to make a wheelchair highly ... device ensures that motorists can see the wheelchair. This ...
(Date:11/23/2014)... Tbdress.com has recently announced that ... Nov. 28, 2014. The special offer will provide ladies many ... one of the world’s largest women’s dresses retailers, and its ... can enjoy great discounts on that day: Everything at Tbdress.com ... (for orders above $89). , People can use the following ...
(Date:11/23/2014)... (PRWEB) November 23, 2014 Locks-Magnetic.com, a ... emergency doors. Moreover, all the new products are available ... The marketing manager is proud of the new emergency ... reset automatically. And customers can choose from several colors. ... Buying high quality emergency door release is not as ...
Breaking Medicine News(10 mins):Health News:Olaparib Industry (CAS 763113-22-0) 2009-2019 Global and China Research Reports is Now Available at RnRMarketResearch.com 2Health News:Olaparib Industry (CAS 763113-22-0) 2009-2019 Global and China Research Reports is Now Available at RnRMarketResearch.com 3Health News:Olaparib Industry (CAS 763113-22-0) 2009-2019 Global and China Research Reports is Now Available at RnRMarketResearch.com 4Health News:Ketogenic Diet is the Perfect Solution for Diet Doc Patients Who Want to Learn How to Lose Weight over the Holidays 2Health News:Ketogenic Diet is the Perfect Solution for Diet Doc Patients Who Want to Learn How to Lose Weight over the Holidays 3Health News:Ketogenic Diet is the Perfect Solution for Diet Doc Patients Who Want to Learn How to Lose Weight over the Holidays 4Health News:Tbdress.com Special Friday Shoes Sales Online Now 2
... red wine, grapes and other fruits, and similar in structure ... fat cells to develop, opening a door to a potential ... Kee-Hong Kim, an assistant professor of food science, and ... in this week,s issue of the Journal of Biological ...
... WEDNESDAY, April 4 (HealthDay News) -- Large amounts of ... doesn,t result in post-traumatic stress disorder -- boosts inflammation ... The findings are important because it,s known that ... to fare worse, according to the researchers at the ...
... Steven Reinberg HealthDay Reporter , WEDNESDAY, April ... to lengthen the lives of patients with a certain ... small phase 2 trial. The drug, belagenpumatucel-L (Lucanix), ... lung cancer and increased the five-year survival rate among ...
... Testing patients with non-small cell lung cancer for both ... may help to predict response to treatment with antifolates, according ... the AACR Annual Meeting 2012, held here March 31 - ... have KRAS gene mutations have a worse prognosis and do ...
... CHICAGO, IL (April 4, 2012)When combined with other treatments, ... growth of cancer cellshas been shown to extend survival ... Unfortunately, about 40 percent of colorectal cancer patientsspecifically ... called KRASdo not respond to the drug. Researchers ...
... To help foster the next generation of global health ... National Institutes of Health are building a network of ... and scientists with a significant mentored research experience in ... will be awarded over the next five years to ...
Cached Medicine News:Health News:Red wine, fruit compound could help block fat cell formation 2Health News:Witnessing, Experiencing Traumatic Events May Worsen Heart Disease 2Health News:Early Study Finds Some Promise for Lung Cancer Vaccine 2Health News:KRAS gene mutation and amplification status affects sensitivity to antifolate therapy 2Health News:Researchers uncover a viable way for colorectal cancer patients to overcome drug resistance 2Health News:NIH awards $20 million over 5 years to train next generation of global health researchers 2Health News:NIH awards $20 million over 5 years to train next generation of global health researchers 3
... Diana System consists of a set of ... main tests conducted in hospital immunohematology labs ... based advanced engineering solutions in robotics, fluidics ... quality of the monoclonal antibodies and the ...
... Resolution - Efficiently separates ... to 1000 bp. It ... PCR fragments, small DNA ... enzyme digestion, and double-stranded ...
... blood grouping reagents for use with ... system is a complete line of ... to determine blood groups in donors ... system includes a negative control for ...
... PK7200 works on patented microplate agglutination technology. ... system for donor ABO/Rh testing. PK7200 also ... and cytomegalovirus antibody detection. The Automated Microplate ... with the proven reliability that for the ...
Medicine Products: