Friday 15 October 2010

Kennedy Krieger Institute: Kennedy Krieger Institute Launches First Phase II Clinical Trial of Heart Disease Treatment for Duchenne Muscular Dystrophy

Kennedy Krieger Institute: Kennedy Krieger Institute Launches First Phase II Clinical Trial of Heart Disease Treatment for Duchenne Muscular Dystrophy: "Kennedy Krieger Institute announced today the launch of a first‐of‐its‐kind, phase II clinical trial to investigate a treatment for heart disease in individuals with Duchenne muscular dystrophy (DMD). Made possible by a $545,000 grant from Pilot Trials Now, an innovative DMD initiative organized and managed by Charley’s Fund Inc. and The Nash Avery Foundation, with additional support from Pfizer Inc., the study will examine whether Revatio (sildenafil) improves cardiac function for those affected by the most common fatal genetic disorder in the world."

Thursday 14 October 2010

Prosensa receives �7.5m milestone payment as part of its program with GlaxoSmithKline in Duchenne Muscular Dystrophy - Prosensa

Prosensa receives �7.5m milestone payment as part of its program with GlaxoSmithKline in Duchenne Muscular Dystrophy - Prosensa: "GSK2402968 (PRO051) is an investigational antisense oligonucleotide which induces exon�skipping of exon 51. The six-month data from the open label Phase IIa extension study in 12�patients with DMD, receiving a weekly 6mg/kg systemic delivery by subcutaneous injection, will�be presented at the 15th International Congress of the World Muscle Society in Japan, October�12-16. The data will be presented by Dr. Nathalie Goemans, MD, from the Department of�Pediatric Neurology, University of Leuven, Belgium."

Acceleron Presents Preliminary ACE-031 Results from a Phase 1 Multiple Ascending Dose Study in Healthy Volunteers

Acceleron Presents Preliminary ACE-031 Results from a Phase 1 Multiple Ascending Dose Study in Healthy Volunteers: "“The preliminary results of this multiple dose study in healthy volunteers confirm and expand upon the encouraging results observed previously in the single dose study of ACE-031,” said Matthew Sherman, MD, Chief Medical Officer of Acceleron. “These data are encouraging as we continue the development of ACE-031 in the ongoing Phase 2 study in patients with�Duchenne Muscular Dystrophy.”"

Monday 11 October 2010

Beaker

Beaker: "Dr. Pier Lorenzo Puri, associate professor at Sanford-Burnham and Italy’s Dulbecco Telethon Institute, and colleagues are figuring out ways to keep the muscle stem cell pool fresh and ready to regenerate injured or diseased muscle. In a study published today in the journal Cell Stem Cell, they uncover the molecular messengers that translate inflammatory signals into the genetic changes that tell muscle stem cells to differentiate. These findings give the scientists a target to artificially dial the stem cell population up or down, a potential treatment that could�boost muscle regeneration in muscular dystrophy patients.

“Our mission is to improve the lives of these patients and extend their lives until they can benefit from a cure 20 years from now,” says Dr. Puri,� a medical doctor who has worked with many muscular dystrophy patients throughout his career."

Antibody that acts as a decoy for mysotatin and binds it before it starts muscle growth inhibition. - Duchennepedia - Action Duchenne - Fighting for a cure for muscular dystrophy

Antibody that acts as a decoy for mysotatin and binds it before it starts muscle growth inhibition. - Duchennepedia - Action Duchenne - Fighting for a cure for muscular dystrophy: "CAMBRIDGE, Mass. – May 5, 2010 – Acceleron Pharma, Inc., a biopharmaceutical company developing novel therapeutics that modulate the growth of cells and tissues including muscle, bone, fat, red blood cells and the vasculature, today announced the initiation of a Phase 2 clinical trial with ACE-031 in patients with Duchenne Muscular Dystrophy (DMD), a fatal neuromuscular disease. ACE-031 is an investigational protein therapeutic that builds muscle and increases strength by inhibiting signaling of a cell surface receptor called the activin receptor type IIB (ActRIIB)."