Navigation Links
PTC Therapeutics Announces Initiation of Phase 2b Registration-Directed Clinical Trial of PTC124 in Duchenne/Becker Muscular Dystrophy
Date:4/23/2008

or the treatment of patients with genetic disorders due to a nonsense mutation, a type of mutation found in approximately 13% of patients with DMD. In this double-blind study, patients will be randomized to receive placebo, or one of two dose levels of PTC124, three times per day. Eligible patients will be boys with nonsense-mutation-mediated DMD/BMD who are at least 5 years of age and are able to walk at least 75 meters or approximately 80 yards in six minutes. PTC expects to enroll a total of 165 patients at approximately 35 investigational sites; all study subjects will undergo 48 weeks of blinded treatment. Thereafter, all participants, including those who have been receiving placebo, will be eligible to enroll in an open-label PTC124 extension study.

The primary outcome measure is the total distance walked during a 6-minute walk test, a test of ambulation that has now been standardized for boys with DMD/BMD through a collaboration with noted investigator, Craig McDonald, M.D., at University of California at Davis. Other outcome measures in the Phase 2b study will evaluate activity at home, muscle and heart function, strength, cognitive ability, muscle integrity, and muscle dystrophin expression. Safety parameters, compliance, and PTC124 blood levels also will be monitored.

"We are very pleased to announce the initiation of the Phase 2b trial for PTC124 in boys with DMD/BMD," said Langdon Miller, M.D., Chief Medical Officer of PTC. "We applaud the patients, parents, and clinicians who have committed themselves to this effort. The design of this trial reflects our ongoing collaboration with the advocacy community, investigators at leading neuromuscular centers, and the U.S. and European regulatory agencies. We hope that PTC124 will soon offer a treatment that addresses the underlying cause of the disease for patients with nonsense-mediated DMD/BMD and that the development of PTC124 will set the stage for improving therapeutic options in this disabli
'/>"/>

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

Page: 1 2 3 4 5

Related medicine technology :

1. Halozyme Therapeutics Presents Findings on Combinations of rHuPH20 Enzyme With Bisphosphonates at the American Association for Cancer Research Conference
2. Halozyme Therapeutics Presents Pre-Clinical Studies of Systemic Delivery of Pegylated rHuPH20 Enyzme in Prostate Cancer Models at American Association for Cancer Research Conference
3. Nektar Therapeutics to Present Positive Data at Upcoming Medical and Scientific Meetings
4. Echo Therapeutics Announces Positive Results with the Prelude(TM) SkinPrep System, the Next-Generation Skin Permeation Medium for its Symphony(TM) Transdermal Continuous Glucose Monitoring System
5. Recent siRNA Research Publications Support Silence Therapeutics Combined Development Approach
6. European Regulatory Agency Accepts Cell Therapeutics, Inc.s Marketing Authorization Application for XYOTAX(TM) for Lung Cancer for Review
7. CV Therapeutics Anti-Ischemic Therapy Ranexa(R) Significantly Reduces Cardiac Chest Pain Symptoms and Recurrent Ischemia in MERLIN-TIMI 36 Patients With Angina
8. Michael J. Fox Foundation Commits up to $10 Million for Pre-Clinical Drug Development Under Therapeutics Development Initiative
9. Synvista Therapeutics Presents New Preclinical Data Demonstrating the Value of Haptoglobin Genotype Testing In Diabetes at ACC Scientific Sessions
10. Cell Therapeutics, Inc. (CTI) Announces Enrollment Complete in Phase III EXTEND (PIX301) Clinical Trial of Pixantrone in Patients With Second or Greater Relapse of Diffuse Large B Cell NHL
11. Dynamis Therapeutics, Inc. Discovers RAGE Inhibitor
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:1/15/2014)... IRVING, Texas , Jan. 15, 2014  Novation, the ... company, awarded several new IT value-added reseller (VAR) national ... with IT goods and services at lower costs. These ... value-added services such as initiative assistance and ongoing IT ...
(Date:1/15/2014)... 2014 Massachusetts General Hospital (MGH), the Broad ... announced today that they have launched a strategic ... targets and develop novel therapies for inflammatory bowel ... worldwide. The MGH-Broad-Amgen collaboration brings together scientists with ...
(Date:1/15/2014)... FORT WORTH, Texas , Jan. 15, 2014  Humberto ... in Dermatology Achievement Award" from the Journal of ... Dermatology Aesthetic & Clinical Conference (ODAC). The event is January ... Florida . The ...
Breaking Medicine Technology:New Novation Information Technology VAR Contracts Offer Significant Savings to Hospitals 2Mass. General, Broad Institute and Amgen Will Work to Discover New Drugs for Inflammatory Bowel Disease 2Mass. General, Broad Institute and Amgen Will Work to Discover New Drugs for Inflammatory Bowel Disease 3Mass. General, Broad Institute and Amgen Will Work to Discover New Drugs for Inflammatory Bowel Disease 4Galderma's global leader honored with a "Lifetime in Dermatology Achievement Award" by Journal of Drugs in Dermatology 2Galderma's global leader honored with a "Lifetime in Dermatology Achievement Award" by Journal of Drugs in Dermatology 3Galderma's global leader honored with a "Lifetime in Dermatology Achievement Award" by Journal of Drugs in Dermatology 4
... 17 Innocoll, Inc., a privately-held,biopharmaceutical company, ... phase,2 clinical trials sponsored by its wholly ... GENTAMICIN TOPICAL for the,treatment and prevention of ... GENTAMICIN TOPICAL is a biodegradable and fully ...
... Biosciences,Incorporated (Nasdaq: KOSN ) presented preclinical data ... in vitro and in vivo,activity as well as ... NEI anticancer mechanism of action correlating inhibition,of the ... cancer cells,compared to normal cells. Data on Kosan,s ...
Cached Medicine Technology:Innocoll Announces Dosing of First Patient in US Phase 2 Clinical Trial to Investigate CollaRx(R) GENTAMICIN TOPICAL for the Treatment of Moderately Infected Diabetic Foot Ulcers 2Innocoll Announces Dosing of First Patient in US Phase 2 Clinical Trial to Investigate CollaRx(R) GENTAMICIN TOPICAL for the Treatment of Moderately Infected Diabetic Foot Ulcers 3Innocoll Announces Dosing of First Patient in US Phase 2 Clinical Trial to Investigate CollaRx(R) GENTAMICIN TOPICAL for the Treatment of Moderately Infected Diabetic Foot Ulcers 4Kosan Presents Preclinical Data on Nuclear Export Inhibitors, Novel Anticancer Agents, at AACR 2Kosan Presents Preclinical Data on Nuclear Export Inhibitors, Novel Anticancer Agents, at AACR 3Kosan Presents Preclinical Data on Nuclear Export Inhibitors, Novel Anticancer Agents, at AACR 4
(Date:4/23/2014)... is looking to give exercise enthusiasts the extra nudge ... shows that a cyber buddy can help. , The ... Journal , is the first to indicate that although ... exercise, a software-generated partner also can be effective. , ... can still motivate people to give greater effort while ...
(Date:4/23/2014)... - Purdue University researchers have developed a way to ... by using tiny gold particles with tails of synthetic ... agricultural and biological engineering, used gold nanoparticles to target ... BRCA1 messenger RNA splice variants, which can indicate the ... these mRNA splice variants in a cell can be ...
(Date:4/23/2014)... of the gastro-intestinal tract has long been considered to ... the Sahlgrenska Academy have used refined microscopic techniques that ... surface of the gastro-intestinal tract is only as large ... , The digestive tract, which passes from the mouth ... a length of about 5 meters in a normal ...
(Date:4/23/2014)... methods that can rapidly screen a large cell ... inside that population has been seriously lacking," said ... the Royal Society of Chemistry journal Chemical ... his coworkers have developed a novel technique that ... Chem. Sci. , 2014, DOI: 10.1039/C4SC00578C, http://pubs.rsc.org/en/content/articlelanding/2014/sc/c4sc00578c#!divAbstract ...
(Date:4/23/2014)... often displayed in the popular Swedish children,s TV ... high-sugar foods. A new study from the University ... children,s TV programmes as well as the link ... weight status. , Steingerdur Olafsdottir,s doctoral thesis focuses ... Swedish children,s TV show Bolibompa. Healthy foods dominated ...
Breaking Medicine News(10 mins):Health News:Cyber buddy is better than 'no buddy' 2Health News:Gold nanoparticles help target, quantify breast cancer segments in a living cell 2Health News:The surface area of the digestive tract 'only' as large as a studio apartment 2Health News:Following a protein's travel inside cells is key to improving patient monitoring, drug development 2Health News:High-calorie and low-nutrient foods in kids' TV 2
... the overweight , , MONDAY, March 3 (HealthDay News) -- ... to have breathing-related problems during surgery, says a University ... looked at 1,380 normal-weight, 351 overweight, and 294 obese ... surgery, found that obese children had a higher rate ...
... WASHINGTON, March 3 To commemorate Patient,Safety Awareness Week ... Epidemiology (APIC) is issuing five steps every,patient can take ... of,developing an infection during a hospital stay., Visit ... learn,more., 1) First Step, Hospital Prep, Most ...
... and advocate for affordable health coverage. Health, labor, faith ... ... Leaders from throughout Michigan,including representatives of insurers, health systems, labor, ... newly formed,Michigan Cover The Uninsured Network (MCTUN). The goal of the ...
... Smith & Nephew,Inc.,s (NYSE: SNN ; ... approval of its award winning OXINIUM(TM),oxidized zirconium technology., ... company to offer such a,revolutionary and award winning ... alloy with a ceramic bearing surface. OXINIUM,provides low ...
... OrthoSynetics(TM), the largest,provider of business services to orthodontic ... Director, IT Infrastructure., "Hiring Brad is in ... and providing the most innovative technology to,our doctors ... CEO,OrthoSynetics(TM). "In a very short period of time, ...
... Super Bowl champ O.J. Brigance,who recently confirmed a ... at The Robert Packard Center for ALS Research ... by the Center to fund,research to find a ... Gehrig,s,Disease.", (Photo: http://www.newscom.com/cgi-bin/prnh/20080303/NEM042 ), ...
Cached Medicine News:Health News:Obese Children Face More Complications During Surgery 2Health News:APIC Issues Recommendations on How to Have a Healthy Hospital Stay 2Health News:Michigan Leaders Join Network to Push for Coverage of State's 1.4 Million Uninsured 2Health News:Smith & Nephew Wins OXINIUM(TM) Approval in Japan 2Health News:Smith & Nephew Wins OXINIUM(TM) Approval in Japan 3Health News:Brad Williams Joins OrthoSynetics(TM) as Director, IT Infrastructure 2Health News:O.J. Brigance Named Honorary Chair of May 3 Race to Cure ALS, Confirms His Diagnosis by The Robert Packard Center for ALS Research at Johns Hopkins 2
Drugs of Abuse (DoA) Array is used for in-vitro diagnostic tests for the qualitative determination of the parent molecule and metabolites of drugs in human urine....
Drugs of Abuse (DoA) Array is used for in-vitro diagnostic tests for the qualitative determination of the parent molecule and metabolites of drugs in human urine....
For use with catalog #0540....
Determination of FDP in Plasma by Latex Agglutination, 60 tests.Microlatex particles coated with mouse monoclonal anti-human FDP antibodies....
Medicine Products: