Category Archives: Muscular Dystrophy Treatment

Sarepta Therapeutics (SRPT) Inc. plummeted the most in 14 months on investor concerns that U.S. regulators wont consider the companys treatment for Duchenne muscular dystrophy on an accelerated basis. Sarepta, with no products on the market, fell 12 percent to $40.95 at 11:11 a.m. The shares of the Cambridge, Massachusetts-based company declined as much as 17 percent, their biggest intraday drop since May 2012, after at least three analysts cut their ratings on the stock. The biotechnology company announced today it plans to seek approval of eteplirsen in the first half of 2013 after the Food and Drug Administration said it was open to a filing based on data from existing studies. The agency requested information on methodology and verification that will be incorporated into the filing, the company said in a statement today. Regulators didnt commit to considering approval of the compound on an accelerated approval basis, Sarepta said. If youre a bear, you believe that the FDA is just saying file, and the management is filing just because they can, not because they think theyre going to get approved, Robyn Karnauskas, a New York-based analyst at Deutsche Bank AG, said in a telephone interview. She reduced her rating … Continue reading →

SOURCE: Sarepta Therapeutics, Inc. CAMBRIDGE, MA--(Marketwired - Jul 23, 2013) - Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced it will hold a conference call at 8:00 a.m. EDT (5:00 a.m. PDT) on Wednesday, July 24, 2013, to provide a regulatory update for eteplirsen, its lead product candidate for the treatment of Duchenne muscular dystrophy (DMD). The conference call may be accessed by dialing 800.446.2782 for domestic callers and 847.413.3235 for international callers. The passcode for the call is 35357086. Please specify to the operator that you would like to join the "Sarepta Therapeutics Regulatory Update Call." The conference call will be webcast live under the events section of Sarepta's website at http://www.sarepta.com and will be archived there following the call for 90 days. Please connect to Sarepta's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary. About Sarepta Therapeutics Sarepta Therapeutics is focused on developing first-in-class RNA-based therapeutics to improve and save the lives of people affected by serious and life-threatening rare and infectious diseases. The Company's diverse pipeline includes its lead program eteplirsen, for Duchenne muscular dystrophy, as well as … Continue reading →

New England Patriots players surrounded her, yet Christine McSherry of Pembroke could not take her eyes off 10-year-old Max as he ran around like a normal kid at a Patriots training camp event last August. Max, like McSherrys own 17-year-old son, Jarrett, suffers from Duchenne muscular dystrophy a disorder, primarily seen in boys, that is caused by a mutation in the gene that codes for the muscle protein dystrophin. Other forms of muscular dystrophy cause a decrease in dystrophin, but boys with Duchenne do not produce any of the protein. Many of them are confined to a wheelchair by age 12. There is no approved treatment for Duchenne muscular dystrophy, which ultimately causes early death. But as McSherry watched the hopping and skipping of Max, a boy whose strength was quickly decreasing a year prior, she felt reason for hope. There was a significant difference, McSherry said Friday. This kid, you would have no idea there was anything wrong with him. The hope comes in the form of eteplirsen, a clinical drug made by Cambridge-based Sarepta Therapeutics. It has shown great potential for stopping the advance of Duchenne. Founder of the Jett Foundation for fighting Duchenne muscular dystrophy, McSherry is … Continue reading →

New England Patriots players surrounded her, yet Christine McSherry of Pembroke could not take her eyes off 10-year-old Max as he ran around like a normal kid at a Patriots training camp event last August. Max, like McSherrys own 17-year-old son, Jarrett, suffers from Duchenne muscular dystrophy a disorder, primarily seen in boys, that is caused by a mutation in the gene that codes for the muscle protein dystrophin. Other forms of muscular dystrophy cause a decrease in dystrophin, but boys with Duchenne do not produce any of the protein. Many of them are confined to a wheelchair by age 12. There is no approved treatment for Duchenne muscular dystrophy, which ultimately causes early death. But as McSherry watched the hopping and skipping of Max, a boy whose strength was quickly decreasing a year prior, she felt reason for hope. There was a significant difference, McSherry said Friday. This kid, you would have no idea there was anything wrong with him. The hope comes in the form of eteplirsen, a clinical drug made by Cambridge-based Sarepta Therapeutics. It has shown great potential for stopping the advance of Duchenne. Founder of the Jett Foundation for fighting Duchenne muscular dystrophy, McSherry is … Continue reading →

by Mike Robinson ELORA Centre Wellington has once again authorized the Elora Fire Fighters Muscular Dystrophy fundraiser on Aug. 24 from 8:30am to 4pm. The toll booth will be at the Metcalfe Street bridge in Elora. Both Wellington County roads committee and police services board support such activities - provided they adhere to requirements of the Safe Streets Act and the Highway Traffic Act. Mayor Joanne Ross-Zuj said that last year, the Elora and Fergus fire stations raised close to $15,000 between two toll booths (one in Elora and one in Fergus). Before approving the fundraiser, councillor Kirk McElwain just wanted to ensure that work on County Road 7 would need to be complete and reopened. The county road was scheduled to close for up to four weeks to deal with work required in the reconstruction of the Elora sewage treatment plant. Councillor Mary Lloyd asked how work on the Tower Street bridge will impact the fundraising efforts of the Fergus firefighters. Typically, that group had held a toll booth on the Tower Street bridge immediately following the Labour Day weekend. Ross-Zuj said she had heard of no alternate plans at this point. Well get that information for you, she … Continue reading →

CAMBRIDGE, Mass., July 15, 2013 /PRNewswire/ --DART Therapeutics Inc., an innovative, new-model biotechnology firm focused on developing therapies for Duchenne muscular dystrophy (DMD), has initiated a phase 1b/2a study of its lead drug candidate, HT-100 (delayed-release halofuginone). The phase 1b study (with a six-month 2a extension) in patients will determine the safety and tolerability of different, increasing doses of HT-100, and explore trends in a range of efficacy endpoints. HT-100 is an orally available, small molecule drug candidate intended to reduce fibrosis and inflammation and promote healthy muscle fiber regeneration in boys with DMD. The phase 1b/2a clinical program will enroll 30 boys and young men with DMD, both ambulatory and non-ambulatory. Participating centers include University of California, Davis Medical Center (Sacramento, Calif.), Kennedy Krieger Institute, Johns Hopkins School of Medicine (Baltimore, MD), Washington University School of Medicine (St. Louis, MO), Cincinnati Children's Hospital Medical Center (Cincinnati, Ohio) and Nationwide Children's Hospital (Columbus, Ohio). DART expects to complete the study in mid 2014. "Treating DMD requires a cocktail of therapies that will change over time," said Gene Williams, CEO of DART Therapeutics. "We believe HT-100 could be a valuable part of this cocktaileven a mini-cocktail on its ownbecause it demonstrates … Continue reading →

Duchenne and Becker Muscular Dystrophy By: Britta Ballerini … Continue reading →

Duchenne and Becker Muscular Dystrophies (DMD and BMD) What are Duchene and Becker Muscular Dystrophies and what causes them? How are they inherited? Diagnosis, symptoms, prognosis, treatment and prevention.CHIP. By: Britta Ballerini … Continue reading →

A MELBOURNE breakthrough has paved the way for cell transplants to grow new muscles and provides hope of overcoming muscular dystrophy and soft-tissue cancers. The world-first discovery also raises the potential to one day regrow tissue lost in serious accidents or convince the body to develop bigger and stronger muscles. A team from St Vincent's Hospital pioneered a method of printing out strands of genetically altered muscle cells combined with a seaweed extract, which they have successfully transplanted into mice and caused them to grow new muscles. The breakthrough could help people with muscular dystrophy and those who have had muscle loss through diseases such as cancer. Lead researcher Assoc Prof Robert Kapsa said the discovery was an exciting step, but warned there was a long way to go before it was known if the treatment would be effective in humans. "We have orientated this work towards things like loss of muscle due to soft-tissue cancers, surgical removal or people who have undergone some sort of trauma, who are able to have a piece of this seaweed substance filled with their own cells, and you can then grow a piece of their muscle back," he said. "What we are looking … Continue reading →

July 7, 2013 Children with Duchenne muscular dystrophy often die as young adults from heart and breathing complications. However, scientists have been puzzled for decades by the fact that laboratory mice bearing the same genetic mutation responsible for the disease in humans display only mild symptoms and no cardiac involvement. Now, researchers at the Stanford University School of Medicine have developed a mouse model that accurately mimics the course of the disease in humans. The study is the first to demonstrate a molecular basis for the cardiac defect that is the primary killer of people with Duchenne muscular dystrophy. Furthermore, the study provides evidence for a potential treatment to help prolong heart function. The mouse model also will allow researchers and clinicians to test a variety of therapies for the inherited condition. "Until now, scientists had no animal model of Duchenne muscular dystrophy that manifests the symptoms of the cardiac disease that kills children and young adults with the condition," said Helen Blau, PhD, the Donald E. and Delia B. Baxter Professor at Stanford and director of the Baxter Laboratory for Stem Cell Biology. "This has been a conundrum for three decades. We found that mice with moderately shortened telomeres … Continue reading →