Navigation Links
PTC Therapeutics Completes Enrollment of Pivotal Trial in Muscular Dystrophy
Date:2/3/2009

encouraged rapid identification of eligible candidates for this important study," said Richard Finkel, MD, attending physician, director of the neuromuscular program, Children's Hospital of Philadelphia. "The completion of this trial enrollment is a striking example of the successes we can achieve when patients, families and the medical community come together to address an area of such high unmet medical need."

"Currently available therapies for nmDMD/BMD may alleviate the symptoms of this progressive debilitating disease, but none address the underlying cause of the disease." said Langdon Miller, MD, chief medical officer of PTC. "Ataluren represents a potential breakthrough as the first treatment designed to enable the formation of functioning dystrophin, the missing protein in patients with nmDMD/BMD."

This is the first pivotal trial for ataluren. In addition, PTC is conducting an open-label, long-term safety study in boys with nmDMD who participated in an early Phase 2a trial. PTC also plans to initiate a second pivotal clinical trial of ataluren in patients with nonsense mutation cystic fibrosis (nmCF) in 2009.

The United States Adopted Names Council (USAN) in conjunction with the World Health Organization, International Nonproprietary Names Committee deemed ataluren's mechanism of action to be significantly novel, warranting creation of a new stem, -luren, for pharmaceuticals that are inducers of ribosomal readthrough of nonsense mutation mRNA stop codons.

"We are pleased to have completed enrollment of our first pivotal study of ataluren in boys and young men with nmDMD/BMD. To have achieved this milestone so far ahead of schedule is a tremendous achievement and we are grateful to the investigators, study coordinators, and the patients and their families for their efforts," said Stuart W. Peltz, PhD, president and CEO, PTC Therapeutics. "We look forward to completing this study ove
'/>"/>

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

Page: 1 2 3 4 5

Related medicine technology :

1. DNA Therapeutics Closes 2.2 Million EUR Series A Financing Round
2. Synvista Therapeutics Announces Termination of Clinical Trials of Alagebrium and SYI-2074 and Provides Business Update
3. Duska Therapeutics Completes Corporate Name Change to Cordex Pharma
4. Phico Therapeutics Receives GBP1 Million Award From the Wellcome Trust to Take Anti-MRSA Nasal Product Through Early Clinical Trials
5. Pixantrone Pre-NDA Communication from FDA Provides Cell Therapeutics Basis to Begin Rolling NDA Submission
6. Cephalon Signs Option Agreement to Acquire Ception Therapeutics
7. Amicus Therapeutics Announces Positive Progress of Three Lead Clinical Programs and General Outlook for 2009
8. Cara Therapeutics Initiates Phase II Proof-Of-Concept Trial of Novel Analgesic in Acute Post-Operative Pain
9. ExonHit Therapeutics Expands Strategic Collaboration With Allergan
10. Synvista Therapeutics Announces Published Data Demonstrating Elevation of Carboxymethyl-Lysine (CML) Is Associated with Decline in Renal Function and Arterial Stiffness in Older Adults
11. Synvista Therapeutics Announces First Dosing of SYI-2074 in Clinical Trial for Psoriasis
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:10/19/2014)... -- Henry Schein, Inc. (NASDAQ: HSIC ), the ... to office-based dental, animal health and medical practitioners, announced ... Inc., a leading full-service provider of dental consumables, implants ... into Japan , the world,s 2 ... the number of countries in which the Company operates. ...
(Date:10/18/2014)... 18, 2014  In a policy paper ... the nation,s largest lesbian, gay, bisexual and transgender ... of Truvada for Pre-Exposure Prophylaxis (PrEP). PrEP is ... prevent the spread of a disease or infection ... name anti-HIV drug combination currently approved for PrEP, ...
(Date:10/17/2014)... 17, 2014  Training market researchers to spot ... factor in today,s highly competitive global marketplace. As ... that effectively and efficiently produce high performing analysts, ... Effective training programs can help ... from internal clients by developing analysts who can ...
Breaking Medicine Technology:Henry Schein Enters Japan With Investment in Iwase Dental Supply 2Henry Schein Enters Japan With Investment in Iwase Dental Supply 3Henry Schein Enters Japan With Investment in Iwase Dental Supply 4Henry Schein Enters Japan With Investment in Iwase Dental Supply 5HRC Endorses PrEP, Calls for Bold Action to Expand Access to Anti-HIV Drug 2HRC Endorses PrEP, Calls for Bold Action to Expand Access to Anti-HIV Drug 3Effective Training Programs for Market Research Groups can Boost Overall Organizational Success 2
... LLC today announced the appointment of Erica L. Mann as ... business based in Morristown, NJ, USA, with operations in more ... million.  Ms. Mann also becomes a member of the Bayer ... 14, 2011. Ms. Mann most recently served as ...
... NEW YORK, Feb. 22, 2011 Reportlinker.com announces ... in its catalogue: North American ... This research covers the state ... and restraints for growth, key trends, and end-user ...
Cached Medicine Technology:Erica L. Mann, Former Pfizer Executive, Appointed New President of Bayer HealthCare's Global Consumer Care Division 2Erica L. Mann, Former Pfizer Executive, Appointed New President of Bayer HealthCare's Global Consumer Care Division 3
(Date:10/20/2014)... Allegheny General Hospital’s (AGH) Cardiovascular Institute ... to repair a damaged mitral valve as the heart ... developed by Abbott Vascular , is designed to ... condition in which the heart’s mitral valve leaflets fail ... the heart’s left ventricle into the left atrium. The ...
(Date:10/20/2014)... October 20, 2014 T.E.N., a ... firm, announced today that William H. Murray, renowned ... 2014 ISE® Luminary Leadership Award winner. Presented annually, ... of an outstanding leader and industry practitioner for ... in advancing the information security industry. , Formerly ...
(Date:10/20/2014)... (PRWEB) October 20, 2014 My Clients ... management software application. The new module enables easy ... receive full life-cycle quality care. , This application allows ... within their practice, attach a to-do item to a ... and even set up reoccurring reminders for follow-ups. ...
(Date:10/20/2014)... Rockynol Retirement Community broke ... 14. The $11 million project will include the latest ... kitchen with full-service restaurant style dining. , “We are ... class Assisted Living apartments,” said Kara Hanzie, Rockynol Executive ... quality of care and this investment is proof of ...
(Date:10/20/2014)... 2014 Final Cut Pro X Plugin developers ... Citrus theme for FCPX filmmakers . , “Fun, cool, ... the Citrus theme” Says Christina Austin, CEO of Pixel Film ... professional.” , Citrus comes with all the tools needed for ... four transitions for added style, a title screen for an ...
Breaking Medicine News(10 mins):Health News:Allegheny General Hospital Introduces New, Beating-Heart Procedure for Mitral Valve Repair 2Health News:Allegheny General Hospital Introduces New, Beating-Heart Procedure for Mitral Valve Repair 3Health News:Bill Murray named 2014 ISE® Luminary Leadership Award Winner 2Health News:Bill Murray named 2014 ISE® Luminary Leadership Award Winner 3Health News:Bill Murray named 2014 ISE® Luminary Leadership Award Winner 4Health News:My Clients Plus Announces New Workflow Management Software 2Health News:Rockynol Retirement Community Expands Assisted Living Center 2Health News:Today Pixel Film Studios Announced the Release of the Citrus theme for Final Cut Pro X 2
... , , SOUTH SAN ... PARD ), a biopharmaceutical company focused on innovative oncology ... executive officer, is scheduled to provide a corporate overview, including ... lead product candidate, picoplatin, at two upcoming investor conferences: , ...
... day cut risk in half in study of Finnish men ... associated with moderate- to high-intensity exercise appears to reduce the ... Finnish study included 2,560 men, aged 42 to 61, whose ... the men had a history of cancer, according to the ...
... CDC recommendations loom, new study confirms this group at great ... officials meet Wednesday to decide who should get priority for ... the virus poses to pregnant women should vault that group ... panel convened by the U.S. Centers for Disease Control and ...
... DARIEN, Conn., July 29 Now served in over 4000 schools across ... is expanding its availability into the retail sector. In addition to ... retailers Tops, D,Agostinos, Big Y, Kings and select Whole Foods ... keeps growing with Wegmans, Price Chopper and Kroger,s Eastern divisions all putting ...
... , , , ... EDI Health Group, Inc., (EHG), a provider of enhanced connectivity, ... in the dental industry, today announced significant additions to its ... during which the company implemented its industry-leading EDI (electronic data ...
... , , JACKSONVILLE, Fla., ... of Representatives for passing a bill (H.R. 3155) that will ... but endorsed a Senate bill (S. 801) that would provide ... caregivers who are in dire need of these services. H.R. ...
Cached Medicine News:Health News:Poniard Pharmaceuticals to Present at Two Upcoming Investor Conferences in August 2Health News:Prioritize Pregnant Women to Get Swine Flu Shot, Experts Say 2Health News:Prioritize Pregnant Women to Get Swine Flu Shot, Experts Say 3Health News:Prioritize Pregnant Women to Get Swine Flu Shot, Experts Say 4Health News:People Across the Country are Making The Switch! 2Health News:EDI Health Group Continues to Drive Growth of EDI Processing Services in the Dental Healthcare Industry Through Addition of 25 New Connections in the First Half of 2009 2Health News:EDI Health Group Continues to Drive Growth of EDI Processing Services in the Dental Healthcare Industry Through Addition of 25 New Connections in the First Half of 2009 3Health News:EDI Health Group Continues to Drive Growth of EDI Processing Services in the Dental Healthcare Industry Through Addition of 25 New Connections in the First Half of 2009 4Health News:Wounded Warrior Project (WWP) Pushes for Senate Bill to Assist Family Caregivers of the Most Severely Injured Veterans 2
Used for dilation of a tract to the kidney for removal of stones. Supplied sterile in peel-open packages. Intended for one-time use....
... via the pelvic nerves has proven to ... incontinence. Through the use of mild electrical ... can be strengthened to encourage urinary control ... to its patients as a tool to ...
... High Dose Rate (HDR) treatment system includes ... Sandwich. Using the Template, simply load ... the Template, suture the Sandwich to the ... the needles in place for the duration ...
... The Prostate Stepper Template Set is the ... dose seed implantation technique. The template set ... that can be sutured to the patient ... with variable ultrasound Grid Face Plates. The ...
Medicine Products: