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PTC Latest News
PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery and development of small-molecule drugs targeting post-transcriptional control processes, today announced positive interim data from a Phase 2 clinical trial of PTC124 in patients with Duchenne muscular dystrophy (DMD) due to a nonsense mutation. The results from the first two cohorts of the three-cohort study show that treatment with PTC124 was associated with increases in muscle dystrophin expression and reductions in serum creatinine kinase values in at least 50 percent of evaluable patients. These data were presented today at the 59th American Academy of Neurology (AAN) Annual Meeting. Read more...   
       
Cardiorespiratoryfunction in the muscular dystrophies
A presentation by Monique Ryan - Children’s Hospital at Westmead
Read more... (PDF 1.13Mb)      
       First US Trial of DMD Gene Therapy Under Way
COLUMBUS, Ohio, March 29, 2006 — The first U.S. human gene therapy trial directed at Duchenne muscular dystrophy (DMD) was launched yesterday at Columbus (Ohio) Children’s Hospital, the Muscular Dystrophy Association (MDA), Children’s Hospital, and Asklepios Biopharmaceutical Inc. (AskBio) announced today. Read more...
New Prospects for treating Muscular Dystrophy
A study on mice suggests that a type of stem cells found in blood vessels may someday be able to regenerate wasting muscle in muscular dystrophy (MD) patients. Read more...
Cardiac MRI at CCHMC
Presentation by William GottliebsonMS MD, Cardiology, CCHMC. Read more... (PDF 730kb)
Consensus statement on the role of glucocorticoid corticosteroids in Duchenne muscular dystrophy
This document reviews the evidence and, presents a protocol of assessment and management for the use of glucocorticoid corticosteroids in Duchenne Muscular Dystrophy (DMD). Read more... (MSWord 174kb)
Steroids/Nutritional Supplements/Antibiotics
There are multiple steroid/supplemental treatments for DMD although there is little agreement (even among researchers and clinicians) about many of them. Read more... (MSWord 40kb)    Exon skipping as a potential therapy for DMD

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