These preliminary results are encouraging and add to the growing body of clinical evidence supporting the potential of PTC124 as a treatment for genetic disorders due to a nonsense mutation, said Stuart W. President and Chief Executive Officer of PTC Therapeutics. The findings in the DMD studies are consistent with the results observed in Phase 2 clinical trials of PTC124 in patients with cystic fibrosis. We intend to expand this concept into other nonsense-mediated genetic disorders.
This Phase 2 multi-site, open-label, is dose – clinical study muscle dystrophin expression in patients with nonsense mutation-mediated DMD. Blood levels of muscle-derived creatine kinase measured as assessments of muscle integrity. PTC124 safety, compliance, and pharmacokinetics are also evaluated. Cincinnati Children’s Hospital Medical Center, Cincinnati, and the University of Utah, Salt Lake City, Utah Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania: – The in the interim in the interim analysis enrolled at three clinical sites in the United States. In the study, patients received 28 days PTC124 treatment at one of two dose levels. All patients were boys with a nonsense mutation in the dystrophin gene, which is substantially elevated serum creatine kinase, and symptoms associated with DMD.
‘All this will be very difficult for the NHS, and we are due to the failure of government, frustrated by the resource Accounting and Budgeting rule which punished in fact respond trust deficit twice.Continue Reading