Navigation Links
PTC Therapeutics Announces Additional Positive Interim Phase 2,Results of PTC124 in Duchenne Muscular Dystrophy

al data, we plan to initiate longer-term clinical trials to evaluate the clinical benefit of PTC124 in patients with DMD."

The Phase 2 clinical trial is being conducted at three sites in the United States: Children's Hospital of Philadelphia, Philadelphia, Pennsylvania; Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio; and the University of Utah, Salt Lake City, Utah. In the study, patients have received 28 days of PTC124 treatment at one of three dose levels. All clinical trial participants are boys with a nonsense mutation in the dystrophin gene, substantially elevated serum creatine kinase levels, and symptoms associated with DMD. The analysis presented today includes data from 26 patients with DMD who received PTC124 at the low-dose and medium-dose levels. Completion of accrual and analysis of data from a higher dose level are ongoing.

The primary endpoint of the trial has been the proportion of patients having an increase in dystrophin expression in muscle during 28 days of treatment with PTC124. Pre- and post-treatment muscle biopsies were available from all 26 patients for analysis. In vitro treatment of patient muscle cells with PTC124 showed evidence of a dose-dependent increase in dystrophin expression in all of the evaluable patients. Preliminary review of the data indicate that, at both dose levels evaluated in this analysis, approximately half of the patients demonstrated visible improvement in the staining for muscle dystrophin in vivo. Overall, four of the six, or 67 percent, of patients treated at the lower dose level and 10 of the 20, or 50 percent, of patients treated at the medium dose level demonstrated an increase in the expression of dystrophin post-treatment. Response did not appear to be dependent on type of nonsense mutation.

Additionally, statistically significant reductions in the concentrations of muscle-derived creatine kinase levels in the blood were observed during PTC124 treatment. Several parents a
'"/>




Page: 1 2 3 4 5

Related medicine technology :

1. Cell Therapeutics, Inc. Management to Discuss Todays Announcement of Interim Pixantrone Study Results
2. Halozyme Therapeutics Enhanze Technology Large Protein Molecule Therapeutic Clinical Trial Results Presented at the 34th Annual Meeting of the Controlled Release Society
3. CV Therapeutics Identifies Potential Anti-Diabetic Mechanism of Action for Ranexa
4. Fat Kills Cancer: Turning Stem Cells from Fat Tissue into Personalized, Cancer-Targeted Therapeutics
5. A 4-Week Therapy with Transition Therapeutics E1-I.N.T. Leads to Sustained Reductions in Blood Glucose Levels for 6 Months Post-treatment in Type 2 Diabetes Patients
6. Clinical Trial Suggests Bone Marrow Stem Cells Are Useful for Spinal Cord Injury; PrimeCell Therapeutics Provided Pre-Clinical Study
7. Opexa Therapeutics Reports Positive Top-line Data in Phase I/II Extension Trial with Tovaxin for Multiple Sclerosis
8. Orexigen Therapeutics to Present Data on Contrave Impact on Visceral Fat and Insulin Resistance at American Diabetes Association 67th Scientific Sessions in Chicago June 23
9. Allos Therapeutics Reports Results for Phase 3 Enrich Study of Efaproxyn in Women With Brain Metastases Originating From Breast Cancer
10. Trophos Publishes ALS Article in Journal of Pharmacology and Experimental Therapeutics
11. Novelos Therapeutics Announces Encouraging Results in Ongoing Phase 2 Ovarian Cancer Trial at Dana-Farber/Partners Cancer Care
Post Your Comments:
(Date:7/11/2014)... -- Kindred Hospital Houston Northwest, with Wound Care Specialists, ... new outpatient wound healing center.  The wound healing ... hard to heal wounds. Chronic wounds include diabetic ... leg wounds related to blood flow issues, post-surgical ... wounds which are complex and demonstrate delayed healing. ...
(Date:7/11/2014)... According to a new market ... (Endoscopes, Endoscopic Operative Devices, and Visualization Systems) - Global ... the global endoscopy devices market was valued at USD ... at a CAGR of 6.8% from 2013 to 2019, ... in 2019. Browse the full Endoscopy Devices Market ...
(Date:7/11/2014)... July 11, 2014 ... Safety & Health Expo 2014 were a ... to London , plus ... exhibition.          (Logo: ... )      (Photo: ...
Breaking Medicine Technology:Kindred Hospital Houston Northwest, With Wound Care Specialists, Opens Outpatient Wound Healing And Hyperbaric Center 2Global Endoscopy Devices Market is Expected to Reach USD 36.9 Billion by 2019: Transparency Market Research 2Global Endoscopy Devices Market is Expected to Reach USD 36.9 Billion by 2019: Transparency Market Research 3Global Endoscopy Devices Market is Expected to Reach USD 36.9 Billion by 2019: Transparency Market Research 4Global Endoscopy Devices Market is Expected to Reach USD 36.9 Billion by 2019: Transparency Market Research 5Global Endoscopy Devices Market is Expected to Reach USD 36.9 Billion by 2019: Transparency Market Research 6UK's Flagship Health & Safety Event Has Successful Inaugural Year in London 2UK's Flagship Health & Safety Event Has Successful Inaugural Year in London 3UK's Flagship Health & Safety Event Has Successful Inaugural Year in London 4
... ALBUQUERQUE, N.M., June 3, 2011 Rep. Ed Whitfield, ... Representatives that will set federal minimum education and certification ... providing, planning and delivering all medical imaging examinations and ... and Excellence ( CARE ) in Medical Imaging and ...
... Diagnostics Incorporated (NYSE: DGX ), the world,s ... that it is scheduled to speak at the Jefferies ... presentation is scheduled for Monday, June 6, 2011 at ... be webcast live during the conference and will be ...
Cached Medicine Technology:Medical Imaging and Radiation Therapy Personnel Certification Bill Introduced in Congress 2Medical Imaging and Radiation Therapy Personnel Certification Bill Introduced in Congress 3
(Date:7/12/2014)... 12, 2014 Sean Francis, owner of the Kawartha ... challenges to his clients. He’s also known for his support of ... a way to combine the two and is excited to announce ... the Boys and Girls Club. , The funds are the result ... June 23rd. Francis sent out emails and asked his clients to ...
(Date:7/12/2014)... prom shopping reaching its final days, VogueQueen.com, a renowned ... its new and trendy apparels. Recently, the company has ... online shop. Additionally, it is now providing huge discounts ... to the company’s sales manager, the promotion will come ... He says, “Now, worldwide clients can order cheap ...
(Date:7/12/2014)... 2014 Doylestown Hospital recently conducted an ... men’s health questions. Paddock tackles topics including main ... women live longer than men, and testosterone replacement therapy, ... Simple featured on Doylestown Hospital’s website, Dr. Brad ... any age. If you notice pain, bulging, or asymmetry ...
(Date:7/12/2014)... July 12, 2014 A recent article released ... a trio of suggestions for those seeking cosmetic surgical ... are rapidly approaching and the heat begins to enliven the ... their summer clothing, realizing that they didn’t work out as ... This can cause a sudden surge of people eager to ...
(Date:7/12/2014)... Los Angeles, CA (PRWEB) July 12, 2014 ... a buyer power score of 4.1 out of ... the market that have been underpinning buyer negotiation ... to IBISWorld procurement analyst Anna Son, “during the ... along with rising costs associated with new product ...
Breaking Medicine News(10 mins):Health News:Kawartha Lakes Fit Body Boot Camp’s Successful Fat Loss Challenge Results in $2,000 to Boys and Girls Club 2Health News:Kawartha Lakes Fit Body Boot Camp’s Successful Fat Loss Challenge Results in $2,000 to Boys and Girls Club 3Health News:VogueQueen.com Updates Its Summer Collection With New And Trendy Evening Dresses 2Health News:Ivyland Medical Center’s Dr. Brad Paddock Offers Tips for Men’s Health 2Health News:Best Summer Cosmetic Surgeries Revealed in Article By Seattle Plastic Surgeon 2Health News:Floor-Grade Instruments Procurement Category Market Research Report from IBISWorld has Been Updated 2Health News:Floor-Grade Instruments Procurement Category Market Research Report from IBISWorld has Been Updated 3Health News:Floor-Grade Instruments Procurement Category Market Research Report from IBISWorld has Been Updated 4
... Build Awareness, Raise,Funds and Help Reduce Premature Births ... 3 JOHNSON,S(R) has announced its,participation in the ... March of Dimes to help build awareness of ... to support the initiatives of the nonprofit,organization. JOHNSON,S(R) ...
... Nov. 3 Avineuro Pharmaceuticals, Inc.,announced today the ... molecule for treatment of Alzheimer,s disease. These studies,demonstrate ... that it has a,favorable pharmacokinetic profile. Based on ... advance AVN-101 into clinical,trials in Q1 2009. Results ...
... Nov. 3 Consumers report significant,interest in online ... survey by StayWell Custom Communications (SCC). However, while ... online videos about health,procedures on their hospital website, ... video., Information on health and wellness on ...
... Celebration with Holiday CD Exclusive, NEW YORK, ... stops,for holiday 2008, a season that includes spectacular ... collaboration that began,this fall continues through the holiday ... recording artists. And, for the,second year in a ...
... - a simple step to manage your health in 2009 , ... ... to manage your health in 2009, is a ground-breaking ill-health prevention strategy ... powerful things people can do to achieve optimal health, live blood tests ...
... WASHINGTON, DC, November 3, 2008 Parents are surprisingly ... visit to their child,s pediatrician, including those who have ... would look to their pediatrician for a referral, according ... the journal Pediatrics . , "This is a ...
Cached Medicine News:Health News:JOHNSON'S(R) and March of Dimes(R) Launch 'Prevent Prematurity Campaign' 2Health News:JOHNSON'S(R) and March of Dimes(R) Launch 'Prevent Prematurity Campaign' 3Health News:Survey Reveals Consumer Interest in New Media Formats for Health Information From Hospitals 2Health News:Survey Reveals Consumer Interest in New Media Formats for Health Information From Hospitals 3Health News:Bloomingdale's Sings 'Oh What Fun' This Holiday Season 2Health News:Bloomingdale's Sings 'Oh What Fun' This Holiday Season 3Health News:Health and Nutrition Diagnostic Tool To Manage Your Health In 2009 2Health News:Health and Nutrition Diagnostic Tool To Manage Your Health In 2009 3Health News:Health and Nutrition Diagnostic Tool To Manage Your Health In 2009 4Health News:Parents comfortable with alcohol screening in pediatricians' offices 2Health News:Parents comfortable with alcohol screening in pediatricians' offices 3
... years, Digisonics has set the standard ... first review station, the only fully ... best multi-modality reporting., ,DigiView cardiology solutions ... workstations, professional reporting choices, an integrated ...
... Cortisol is a glucocorticoid produced by the ... (ACTH). It is secreted with a circadian ... in the morning. Cortisol is often elevated ... hypertension, stress, AIDS, and in the visceral ...
... immunoassay system built around the many positive ... important addition of Logic Driven Incubation (LDI). ... for optimal performance with the fastest possible ... such as the cardiac biomarkers and HCG ...
... System from KUBTEC is a dual capability system ... dimensional representation of the specimen under test. While ... X-ray images, the micro focus source also allows ... in standard 2D images. The 100 mm x ...
Medicine Products: