Navigation Links
PTC Therapeutics Initiates Registration-Directed Phase 3 Trial of Ataluren in Cystic Fibrosis
Date:9/10/2009

uren (PTC124(R))

Ataluren is the first investigational new drug designed to restore the formation of a functioning protein in patients with genetic disorders due to a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. Ataluren is currently being investigated for use in patients with nmCF and nmDMD/BMD.

Ataluren has been granted orphan drug status for the treatment of nmCF and nmDMD/BMD by the U.S. Food and Drug Administration (FDA) and the European Commission. The FDA has also granted ataluren Subpart E designation for expedited development, evaluation, and marketing. The development of ataluren has been supported by the Cystic Fibrosis Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic Fibrosis Foundation), the FDA Office of Orphan Products Development, the Muscular Dystrophy Association, Parent Project Muscular Dystrophy, and the National Center for Research Resources.

Completed Ataluren Clinical Trials

Data from Phase 2a clinical trials of ataluren in pediatric and adult patients with nmCF show that administration of ataluren results in production of functional CFTR and statistically significant improvements in CFTR chloride channel function in the airways. Ataluren treatment is also associated with reductions in cough frequency and improvements in pulmonary function tests.

Across all ataluren clinical trials to date, including Phase 1 healthy-volunteer trials, ataluren has been generally well tolerated. In Phase 2a trials in nmCF and nmDMD/BMD, adverse events have been largely consistent with background symptoms and have usually been mild. No concerning adverse findings have been identified based on physical examinations, vital sign measurements, electrocardiograms, or laboratory studies. The mean compliance with ataluren therapy has been greater than 90 percent in all
'/>"/>

SOURCE PTC Therapeutics, Inc.
Copyright©2009 PR Newswire.
All rights reserved

Page: 1 2 3 4 5 6

Related biology technology :

1. Vicus Therapeutics to Present at the 234th American Chemical Society National Meeting in Boston, MA
2. DJO Incorporateds Pending Merger With ReAble Therapeutics Clears U.S. Antitrust Review
3. PeriCor Therapeutics Announces Closing of Licensing Agreement for Acadesine with Schering-Plough Corporation
4. Systems Medicine (SM), a Subsidiary of Cell Therapeutics, Inc. (CTI), Announces Execution of CRADA with NCI/NIH to Develop Potential Cancer Treatments Targeting the Insulin Growth Factor (IGF) Pathway
5. Amsterdam Molecular Therapeutics to Release H1 2007 Results on August 29, 2007
6. Nektar Therapeutics Appoints Tim Harkness as Chief Financial Officer
7. ExonHit Therapeutics: Allergan and ExonHits First Collaboration Compound to Begin Human Clinical Trials
8. Cell Therapeutics, Inc. Takes Corrective Actions in Response to Nasdaq Determination
9. Amsterdam Molecular Therapeutics Reports Half Year Results 2007
10. Nektar Therapeutics Appoints Lutz Lingnau as New Board Member
11. Anesiva Grants Specific-Use License of Its Needle-Free Drug Delivery Technology to Particle Therapeutics for Diabetes Drug
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:1/22/2015)... January 22, 2015 GEA Niro Soavi ... top laboratory homogenizer, the PandaPLUS 2000, which is ideal ... , and cell disruption . This compact laboratory ... fruit juices, liquid food, food additives and ingredients as ...
(Date:1/22/2015)... 22, 2015 Crystal Diagnostics (CDx) Xpress System, ... has received AOAC-PTM Certifications for the six non-O157 Shiga toxin-producing ... collectively referred to as STEC or the “Big-6”) as well ... unit (cfu) per 325 g of raw ground beef and ...
(Date:1/22/2015)... 2015 Selexis SA, a serial innovation ... Banks (RCBs) used for drug discovery to commercial manufacturing, ... include Next-Generation Sequencing (NGS) data packages. The ... manufacturing by ensuring the integrity of the gene, validation ...
(Date:1/22/2015)... CAMBRIDGE, Mass. , Jan. 22, 2015   GenoSpace , ... suite of tools to enable the broad use of genomic, ... today announced the appointment of Michelle Munson , CEO ... independent director. Logo - http://photos.prnewswire.com/prnh/20150122/170713 ...
Breaking Biology Technology:GEA Niro Soavi Announces the PandaPLUS 2000 Homogenizer for Processing New Applications for Nanoemulsions and Cell Disruption 2Crystal Diagnostics Awarded AOAC-PTM Accreditation for the Rapid Detection of “Big 6” E.coli Food Pathogens 2Selexis Generated Research Cell Banks Now Fully Sequenced Using Next-Generation Sequencing 2GenoSpace Expands Board with Appointment of Michelle Munson 2
... , ... Group is pleased to announce that Dr. Michael Langley is joining the company as ... served many roles during his career with Lilly in both animal science and human ... led a number of successful regulatory engagements including both European and US regulatory submissions. ...
... , WINSTON-SALEM, N.C. , Jan. 29 ... They are the stars of a new animated film, "Cell Side ... in cooperation with Wake Forest University and other community ... and female animated characters that dramatize what happens within cells. ...
... Jan. 28 Tissue Genesis, Inc., an emerging leader ... Medical Systems Holdings, Inc. (Nasdaq: AMMD ) announced today ... of adipose (fat)–derived regenerative cells for use in treating pelvic ... over the life of the agreement to obtain rights ...
Cached Biology Technology:Michael Langley, DVM, MBA, RAC Joins the Anson Group as a Regulatory Consultant 2New Musical 'Cell Side Story' Puts Science on Stage 2New Musical 'Cell Side Story' Puts Science on Stage 3American Medical Systems and Tissue Genesis Sign $1.1 Million Collaboration Deal 2American Medical Systems and Tissue Genesis Sign $1.1 Million Collaboration Deal 3American Medical Systems and Tissue Genesis Sign $1.1 Million Collaboration Deal 4
(Date:12/17/2014)... Valencell, a leader in performance biometric data sensor technology, ... such as Intel, Jabra, Atlas, and Scosche to develop ... products will be showcased at CES 2015, taking place ... "Our partners choose PerformTek biometrics, because ...
(Date:12/11/2014)... , Dec. 09, 2014 Research and Markets ... ( http://www.researchandmarkets.com/research/9ql3kr/biometrics_market ) has announced the addition of the ... their offering. One major trend emerging ... systems. Multimodal biometric systems utilize more than one characteristic ...
(Date:12/10/2014)... Forest Baptist Medical Center today announced plans for a new ... this $50 million capital project is part of a larger ... The medical education building will be located in ... to 525@vine in Wake Forest Innovation Quarter. Construction will begin ...
Breaking Biology News(10 mins):Valencell PerformTek Biometrics Power the Most Accurate Wearables at CES 2015 2Biometrics Market in India 2015-2019: Key Vendors are 3M Cogent, NEC, Safran and Suprema 2Wake Forest Baptist to Build New Medical Education Facility In Wake Forest Innovation Quarter 2Wake Forest Baptist to Build New Medical Education Facility In Wake Forest Innovation Quarter 3Wake Forest Baptist to Build New Medical Education Facility In Wake Forest Innovation Quarter 4
... the American Society of Tropical Medicine and Hygiene, the ... on research advances in the fight against diseases that ... in Central Florida, animal-borne diseases the world over and ... innovations to combat the global epidemic of counterfeit malaria ...
... developed a strain of the Middle East respiratory syndrome coronavirus ... disease, according to a study to be published in ... for Microbiology. The mutant MERS virus, rMERS-CoV-ΔE, has a mutation ... a cell and replicating its genetic material, but deprives it ...
... , Sept. 9, 2013 /PRNewswire-iReach/ -- BioForce Nanosciences, Inc. ... international network of partners and distributors. The partnership will help ... Europe , the Middle East ... Asia . BioForce specializes in nanometer-scale ...
Cached Biology News:American Society of Tropical Medicine and Hygiene 62nd Annual Meeting Nov. 13-17, Washington DC 2American Society of Tropical Medicine and Hygiene 62nd Annual Meeting Nov. 13-17, Washington DC 3Scientists engineer strain of MERS coronavirus for use in a vaccine 2Scientists engineer strain of MERS coronavirus for use in a vaccine 3BioForce Nanosciences, Inc. Expands Its Global Market. New partnership with Acugenex Limited 2
... 1,4-Dithiothreitol DTT ... Purity: ≥99% by titration. ... (100 mM, 1 cm, 260 nm): ≤0.500; ... Heavy metals: ≤0.001%. Soluble ...
... Basically, most ``special`` dye stains are ... few other non-routine) stains are $9.00. ... a paraffin block, staining, coverslipping and ... (for senile plaques) ,Bodian`s Stain ,Copper ...
... Dithiothreitol DTT White solid.. PACKAGED ... to thiols, so reaction is "driven" to completion. ... contain waxy surfactant found in many other preparations. ... Purity: ≥97% by titration. Contaminants: ...
... This kit is designed for easy ... The protocol allows for successful transformation simply ... kit reagents with cultured yeast cells. No ... washing, are required. The kit is particularly ...
Biology Products: