Monday, November 26, 2012 Sarepta Therapeutics (Nasdaq:SRPT) says its collaborating with a London scientist and other organizations for development of a fourth drug candidate for the treatment of Duchenne muscular dystrophy (DMD), a rare, degenerative neuromuscular disorder causing muscle loss and a premature death. The move is spurred in part by good results of a Phase 2b study announced last month of eteplirsen, Sareptas lead drug candidate using its proprietary technology to skip a sequence of DNA, known as exon 51, which is hoped to enable the repair of genetic mutations that affect about 13 percent of the total DMD population. The results of the trial showed significant improvement over a placebo, according to the company, in a 48-week Phase 2b study measuring increase in dystrophin, a protein which slows the progression of the disease, as well as the so-called six minute walk test, measuring the distance a DMD patient can walk in six minutes. The aim of the new collaboration - with professor Francesco Muntoni, M.D., of the University College London, the Dubowitz Neuromuscular Centre, the Institute of Child Health and other scientists from the European Union and United States- is to develop a drug to similarly skip another … Continue reading →

A SPECIALIST physiotherapy unit is opening in Coventry to help people with muscular dystrophy. The neuromuscular centre at Herewood College in Tile Hill will be the first of its kind in the region and only the second in the country. Charity chiefs will join campaigners to launch the centre this week. There are more than 3,000 people who suffer from the muscle wasting condition in Coventry who could benefit from the new service. Coun Harjinder Singh Sehmi, whose daughter is one of those patients, is backing the centre. He said: "My daughter has been to [the first centre] in Cheshire twice and I proposed they should have a centre in the Midlands as well. "I was delighted when they told me they were already planning one. "This will be very beneficial to the community of Coventry." Muscular dystrophy is an inherited disease that affects an estimated 11,500 people in the Midlands. As the condition progresses, patients often have to use a wheelchair or even a ventilator. More here: Specialist centre to open in Coventry to help those with muscle wasting diseases … Continue reading →

CAMBRIDGE, MA--(Marketwire - Nov 26, 2012) - Sarepta Therapeutics ( NASDAQ : SRPT ), a developer of innovative RNA-based therapeutics, announced today a collaboration for the development of an additional exon-skipping drug targeting exon 53, its fourth drug in development, in support of Sarepta's broad-based program for the treatment of Duchenne muscular dystrophy (DMD).Sarepta's collaboration is with University College London's (UCL) scientist, Professor Francesco Muntoni, MD, the Dubowitz Neuromuscular Centre, the Institute of Child Health and other scientists from the EU and US.The EU Health Innovation-1 2012 Collaborative research grant will support certain IND-enabling activities and clinical proof of concept studies for an exon 53-skipping therapeutic.Sarepta recently announced positive results from its extension study of its Phase IIb trial of eteplirsen, its exon 51-skipping therapeutic candidate for the treatment of DMD.Sarepta is also developing other PMO-based exon-skipping drug candidates for exons 45 and 50. "The recent compelling clinical data on eteplirsen targeting exon 51, which started with our work on the Phase I study in the UK, provides a strong foundation for using Sarepta's technology against exon 53," said Francesco Muntoni, professor of pediatric neurology and head of the Dubowitz Neuromuscular Centre at the UCL Institute of Child Health, London. … Continue reading →

Tuesday, November 27, 2012 Finding a treatment for Duchenne muscular dystrophy (DMD) wont just mean developing one drug, says Chris Garabedian, president and CEO of Sarepta Therapeutics (Nasdaq:SRPT). It will mean developing as many as 20. The fatal inherited disorder affects one in 3,500 young boys, confining most of them to wheelchairs by the age of 12. Few patients with it live past the age of 30. Years of telethons by the Muscular Dystrophy Association and comedian Jerry Lewis have raised awareness of the disease, but today, available treatments are mostly limited to steroids - which only prolong the boys ability to walk by 2-5 years - and surgery, including heart transplants, according to the National Human Genome Institute. After Sareptas announcement this week that it will begin development of a fourth drug candidate for the treatment of DMD, Garabedian told Mass High Tech that the company is close to understanding the very origins of the disease in mutations of one of the longest genes in the human body, which is responsible for creating the muscle protein, dystrophin. Seraptas approach is to develop drugs which skip certain sections of the DNA where mutations inhibit production of dystrophin. While that wouldnt … Continue reading →

Public release date: 27-Nov-2012 [ | E-mail | Share ] Contact: Paddy Moore padmoore@ohri.ca 613-737-8899 x73687 Ottawa Hospital Research Institute November 27, 2012 Ottawa Scientists have discovered that injecting a novel human protein into muscle affected by Duchenne muscular dystrophy significantly increases its size and strength, findings that could lead to a therapy akin to the use of insulin by diabetics. These results were published today in the Proceedings of the National Academy of Sciences by Dr. Julia von Maltzahn and Dr. Michael Rudnicki, the Ottawa scientist who discovered muscle stem cells in adults. "This is an unprecedented and dramatic restoration in muscle strength," says Dr. Rudnicki, a senior scientist and director for the Regenerative Medicine Program and Sprott Centre for Stem Cell Research at the Ottawa Hospital Research Institute. He is also a Canada Research Chair in Molecular Genetics and professor in the Faculty of Medicine at the University of Ottawa. "We know from our previous work that this protein, called Wnt7a, promotes the growth and repair of healthy muscle tissue. In this study we show the same types of improvement in a mouse model of Duchenne muscular dystrophy. We found that Wnt7a injections increased muscle strength almost two-fold, … Continue reading →