Navigation Links
PTC Therapeutics Announces European Medicines Agency Validation of Marketing Authorization Application for Ataluren in Duchenne Muscular Dystrophy
Date:12/6/2012

>

PTC also is currently conducting ongoing open-label safety and tolerability studies of ataluren for patients who were in previous ataluren clinical trials for nmDMD in both the United States and Europe. The company has worldwide development and commercial rights for ataluren in all indications and territories. Ataluren is also being developed for nonsense mutation cystic fibrosis and PTC is in discussions with regulators regarding next steps.

About Conditional Approval
Conditional approval is granted based on a positive benefit/risk ratio in the available data which, while not yet comprehensive, indicate that the public health benefits of immediate availability of a medicine outweigh its risks. The company is given obligations to fulfill, such as the performance of further studies. The approval is renewed on a yearly basis until all obligations have been fulfilled, and is then converted from a conditional approval into a full approval. Conditional approvals can only be granted for medicines that satisfy an unmet medical need, meaning the medicine is intended to be used for a disease or condition for which no treatment is readily available, and it is therefore important that patients have early access to the medicine concerned.

About Duchenne Muscular Dystrophy (DMD)
Primarily affecting males, Duchenne muscular dystrophy (DMD) is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of skeletal, diaphragm, and heart muscles. Patients with Duchenne muscular dystrophy, the more severe form of the disorder, lose the ability to walk as early as age 10 and experience life-threatening lung and heart complications in their late teens and twenties. There are an estimated 35,000 patients with DMD in the United States and Europe and approximately 13 percent of all DMD cases are caused by nonsense mutations in the dystrophin gene. More information about DMD is available th
'/>"/>

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

Page: 1 2 3 4 5

Related medicine technology :

1. Cell Therapeutics, Inc. (CTI) to Report First Quarter Financial Results on April 20
2. Faruqi & Faruqi, LLP Encourages Investors Who Suffered Substantial Losses Investing in Chelsea Therapeutics International, Ltd. to Contact the Firm
3. Argos Therapeutics Secures $25 million Series D Financing to Commence Phase 3 ADAPT Study in Patients with Metastatic Renal Cell Carcinoma (mRCC) in Mid-2012
4. Novelos Therapeutics Announces Successful Completion of First Cohort In Lung Cancer Trial With I-124-CLR1404 (LIGHT) Cancer-Targeted PET Imaging Agent at UW Carbone Cancer Center
5. Zacks Industry Outlook Highlights: Eli Lilly, Forest Labs, ViroPharma, Novartis and United Therapeutics
6. Oxford BioTherapeutics Appoints Senior Industry Leader to Head its New Clinical Development Operations in Basel, Switzerland
7. Nektar Therapeutics Reports Financial Results for the First Quarter of 2012
8. Cara Therapeutics Licenses Novel Kappa Opioid Agonist, CR845, to Chong Kun Dang Pharmaceutical Corporation For South Korean Market
9. Frost & Sullivan: High Uptake of New Drug Therapies and Aging Population Expansion Keep the U.S. Retinal Therapeutics Market Buoyant
10. Zenobia Therapeutics, Inc. And BioBlocks, Inc. Announce Sale Of A Joint Fragment Library Ideal For Screening By All Popular Methods
11. New Forecast Shows Global Protein Therapeutics Market to Reach $143.4 Billion by 2015
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:5/6/2015)... SAN FRANCISCO , May 6, 2015 /PRNewswire/ ... (MZEI:OB, MZEI:QB), manufacturers of the AsepticSure® Hospital Disinfection ... indicators used to confirm medical instrument sterilization in ...   absolute bactericidal kill for hospital room disinfection. ... process indicators that can directly monitor the lethality ...
(Date:5/6/2015)... -- Eli Lilly and Company (NYSE: LLY ) ... and device innovation center in Cambridge, Massachusetts ... attract top scientists and bioengineers, as well as enhance ... Lilly Cambridge Innovation Center, a makerspace located in Kendall ... to explore how emerging technologies and connectivity can advance ...
(Date:5/6/2015)... , May 6, 2015   Sigma-Aldrich Corporation (NASDAQ: ... technology company, today announced a $50,000 product donation to ... initiative to replicate key findings from recent high-impact papers ... a collaboration between the online research marketplace Science ... and the open-access journal eLife. The ...
Breaking Medicine Technology:Hospital Rooms Cleaned to Autoclave Standards 2Eli Lilly and Company Reveals Plan for Innovation Center in Cambridge, Massachusetts 2Eli Lilly and Company Reveals Plan for Innovation Center in Cambridge, Massachusetts 3Sigma-Aldrich Donates $50K in Products to the Reproducibility Project to Foster Replicable Research and Accelerate Scientific Discoveries in Cancer Biology 2Sigma-Aldrich Donates $50K in Products to the Reproducibility Project to Foster Replicable Research and Accelerate Scientific Discoveries in Cancer Biology 3
... BURLINGTON, Vt., Feb. 6, 2012  After only three years ... Award from the International Society for Optics and Photonics ... products in the category of  "Life Sciences and Biophotonics." ... a patent-pending, innovative, high-intensity and exceptionally stable new light ...
... Pharmaceuticals, Inc. (NASDAQ: CADX ), a biopharmaceutical ... principally for use in the hospital setting, announced today ... (acetaminophen) injection. Cadence has notified the U.S. Food and ... recall of OFIRMEV lot number V005710 was initiated due ...
Cached Medicine Technology:89 North Wins Prestigious International Award 2Cadence Pharmaceuticals Announces Voluntary Recall of One Lot of OFIRMEV® (Acetaminophen) Injection 2Cadence Pharmaceuticals Announces Voluntary Recall of One Lot of OFIRMEV® (Acetaminophen) Injection 3Cadence Pharmaceuticals Announces Voluntary Recall of One Lot of OFIRMEV® (Acetaminophen) Injection 4
(Date:5/6/2015)... May 06, 2015 The Florida Council ... Health Coalition, Group Victory, Miami Area Geriatric Education Center, ... of Broward County Commission on Substance Abuse, will hold ... May 12-14, at the Riverside Hotel. , This important ... education for everyone as well as clinical training for ...
(Date:5/6/2015)... york, NY (PRWEB) May 06, 2015 ... strategy, design, creative and technology, announced today that its ... expert advisor by Shocase, a marketing professional network aimed ... marketing sector. , Widely regarded as a specialist in ... the most high-profile branding programs over the last three ...
(Date:5/6/2015)... In celebration of their twentieth anniversary, Premierlacewigs.com launched ... away a deluxe human hair lace wig valued at $300 ... on a third-party giveaway tool. The important thing is the ... winner. , The hair is a crown, a reflection of ... a jewel for the past 20 years. Whether it is ...
(Date:5/6/2015)... May 06, 2015 Most people in the ... nursing or other care after age 65. But less than ... for it or even talking about it, reports the May ... people risk two problems. One is losing control over the ... families," says Dr. David Grabowski, a professor of health care ...
(Date:5/6/2015)... 06, 2015 Doctors who have done ... they are calling “unprecedented” tumor control in mesothelioma patients. ... new research. Click here to read it now. ... 25 mesothelioma patients between March and December 2014. They ... , “Pembrolizumab… provides robust antitumor activity in patients with ...
Breaking Medicine News(10 mins):Health News:Conference on Problem Gambling to be held in Ft. Lauderdale, FL 2Health News:Shocase Selects MBLM’s Claude Salzberger as Company Advisor 2Health News:PremierLaceWigs.com Human Hair Lace Wigs Giveaway Could Change Your Hairstyle 2Health News:Start Planning Now for Long-Term Care, from the May 2015 Harvard Health Letter 2Health News:Researchers Say “Unprecedented” Mesothelioma Response Possible with Skin Cancer Drug, According to Surviving Mesothelioma 2
... Scientists at Warwick Medical School have uncovered the molecular ... ,health checkpoint, with cells that carry unequal numbers of ... , Studying simple yeast cells, scientists now understand the ... the correct number of chromosomes. , Most ...
... State University will lead a four-year, $5 million expanded ... discover proteins, or biomarkers, to help predict cardiovascular disease ... type 2 diabetes. Nearly 26 million Americans have ... U.S. The disease is a major cause of heart ...
... , TUESDAY, Nov. 1 (HealthDay News) -- Multiple sclerosis ... aim at the abnormal behavior of a specific type of ... the cells in question -- known as "B cells" -- ... and disabling nervous system disease, commonly called MS. The ...
... high school students are considered overweight, and while a myriad ... parents can do to help curb these rates, very little ... lose weight. Research led by public health doctoral candidate ... Philadelphia report wanting to lose weight, their actions are more ...
... an eight hour shift leads to substantial cost savings and energy ... Journal of the American College of Radiology . ... the use of computers and scanning equipment. "We should ... this equipment and look for ways to reduce it, both as ...
... NYU Langone Medical Center identified for the first time that ... outer and inner layers of the brain, called "blurring", may ... development of new therapies for degenerative brain conditions, such as ... issue of the Journal of Neuroscience . ...
Cached Medicine News:Health News:Warwick scientists discover how daughter cells receive the same number of chromosomes 2Health News:ASU leads $5 million NIH-sponsored research initiative to advance diabetes care and treatment 2Health News:ASU leads $5 million NIH-sponsored research initiative to advance diabetes care and treatment 3Health News:New Multiple Sclerosis Therapy Promising in Early Trial 2Health News:New Multiple Sclerosis Therapy Promising in Early Trial 3Health News:Study finds overweight teens want to lose weight, going about it the wrong way 2Health News:Neurologists identify potential biomarker of cognitive decline for earlier diagnosis of disease 2
Illuminated Phoroptor Refracting Instrument. Illumination is provided by a cool-running, energy efficient LEDs (Light Emitting Diodes) that light only what you need to see without affecting the darke...
Occluder and Maddox Rod...
Graham Field 10' Reversed Snellen Eye Chart....
Ultrasonic Pachymeter UP-1000 is fast, accurate corneal thickness measurements....
Medicine Products: