Category Archives: Muscular Dystrophy Treatment

Public release date: 9-Aug-2013 [ | E-mail | Share ] Contact: Gina Bericchia Gina.Bericchia@NationwideChildrens.org 614-355-0495 Nationwide Children's Hospital New research on two promising gene therapies suggests that combining them into one treatment not only repairs muscle damage caused by Duchenne muscular dystrophy, but also prevents future injury from the muscle-wasting disease. The work, led by a team at The Research Institute at Nationwide Children's Hospital, is the first to look at the approach in aged mice, a key step toward clinical trials in patients. The findings were published in July in Human Molecular Genetics. "We're excited about the fact that these are older mice and we're still able to see a sustained functional benefit from this combined therapythis hasn't been shown before," says Louise Rodino-Klapac, PhD, a principle investigator in the Center for Gene Therapy at Nationwide Children's and lead author of the research. Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy in children, affecting about one out of every 3,500 to 6,000 male births. Symptoms usually begin between the ages of 3 and 5 years and progress rapidly. Most patients are unable to walk by age 12 and ultimately need a respirator to breathe. Patients … Continue reading →

Aug. 10, 2013 Results from a clinical trial of eteplirsen, a drug designed to treat Duchenne muscular dystrophy, suggest that the therapy allows participants to walk farther than people treated with placebo and dramatically increases production of a protein vital to muscle growth and health. The study, led by a team in The Research Institute at Nationwide Children's Hospital, is the first of its kind to show these results from an exon-skipping drug -- a class of therapeutics that allows cells to skip over missing parts of the gene and produce protein naturally. "I've been doing this for more than 40 years and this is one of the most exciting developments we've seen," says Jerry Mendell, MD, lead author of the study and director of the Center for Gene Therapy at Nationwide Children's. "It offers great hope to patients with Duchenne muscular dystrophy and their families." The research, which appears online Aug. 1 in the journal Annals of Neurology, is the first study from a double-blind controlled randomized trial of an exon-skipping agent to provide conclusive proof based on the standard six-minute walk test used to measure muscle function in patients with Duchenne muscular dystrophy (DMD), the most common form … Continue reading →

Additional US patent is important for the company, CEO says Glyn Edwards, CEO of drug discovery company Summit, tells Proactiveinvestors the additional patent for Duchenne Muscular Dystrophy treatment C1100 the compan... By: ProactiveInvestors … Continue reading →

CAMBRIDGE, MA--(Marketwired - Aug 8, 2013) - Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today reported financial results for the three months and six months ended June 30, 2013, and provided an update of recent corporate developments. "We are excited that the FDA is open to an NDA filing for our drug eteplirsen for the treatment of Duchenne muscular dystrophy and the organization is focused on all of the activities necessary for a successful NDA submission in the first half of next year," said Chris Garabedian, president and chief executive officer of Sarepta Therapeutics. "The Sarepta team is planning for success as we continue activities related to our eteplirsen confirmatory study and our manufacturing scale up, while we begin pre-commercial activities to prepare for the potential approval of eteplirsen." Financial Results For the second quarter of 2013, Sarepta reported a Non-GAAP net loss of $14.6 million, or $0.46 per share, compared to a Non-GAAP net loss of $5.0 million for the second quarter of 2012, or $0.22 per share. The incremental loss is primarily the result of an $8.3 million decrease in government contract revenues as well as a $1.2 million increase in Non-GAAP operating expenses. … Continue reading →

Results Show a Significant Increase in Dystrophin Production and a Stabilization of Walking Ability in Duchenne Muscular Dystrophy Patients CAMBRIDGE, MA--(Marketwired - Aug 8, 2013) - Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced the first peer-reviewed publication of the 48-week results from the Phase IIb clinical study of eteplirsen in the Annals of Neurology. Eteplirsen is an investigational medicine in development for the treatment of patients with Duchenne muscular dystrophy (DMD) who have a genotype amenable to skipping of exon 51. Published study results showed that once-weekly treatment with eteplirsen resulted in a statistically significant increase from baseline in novel dystrophin, the protein that is lacking in patients with DMD. In addition, eteplirsen-treated patients evaluable on the 6-minute walk test (6MWT) demonstrated stabilization in walking ability compared to a placebo/delayed-treatment cohort. Eteplirsen was well tolerated in the study with no clinically significant treatment-related adverse events. These data will form the basis of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for eteplirsen planned for the first half of 2014. "These unprecedented data for eteplirsen in DMD patients with genetic mutations correctable by skipping exon 51 represent a significant milestone … Continue reading →

Lexington, Massachusetts (PRWEB) August 06, 2013 Today, the FSH Society, a Massachusetts based non-profit that is a world leader in combating facioscapulohumeral dystrophy (FSHD), announced that it has awarded a $48,909 research grant to Dr. Jeffrey Statland, University of Rochester Fellow and Senior Instructor in the Department of Neurology. The grant will fund Dr. Statlands evaluation of electrical impedance myography (EIM) as a tool for quantifying the muscle structure of FSHD patients. EIM offers a portable, quick and easy way to measure muscle changes in FSHD patients. The EIM tool was developed by Seward Rutkove, MD, and is manufactured by Convergence Medical Devices which is based in Boston, Massachusetts. FSHD, one of the most prevalent types of muscular dystrophy, is a degenerative muscle disease that causes progressive weakness, usually starting with the face, shoulder and arms, but which can affect almost any skeletal muscle. FSHD affects approximately 500,000 people worldwide and between one and two percent of the population carries a genetic trait that places future generations at risk of the disease. Currently, there is no treatment. We are thrilled that the FSH Society continues supporting the development of new clinical outcomes for future FSHD clinical trials, said Dr. Statland. … Continue reading →

PEMBROKE 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 McSherry's 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 recently. "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 … Continue reading →

NEWPORT BEACH, Calif.--(BUSINESS WIRE)-- Ryan Getzlaf, captain of the Anaheim Ducks, is hosting the third annual Getzlaf Golf Shootout on September 8 to benefit CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy. Gold medalist and NHL All-Star Getzlaf, along with his Anaheim Ducks teammates including Corey Perry, Dustin Penner and Sauk Koivu, and other hockey players, celebrities and community leaders will participate in the golf tournament to support CureDuchenne. Scott Niedermayer, assistant coach of the Anaheim Ducks, Stanley Cup champion, Olympic gold medalist and Hockey Hall of Fame inductee, is also scheduled to participate. This charity event will be held at the Monarch Beach Golf Links in Dana Point on Sunday, September 8. Each foursome will be teamed up with a professional athlete or celebrity for a unique and exciting day on the green. A pre-golf dinner reception will be held at Sutra in Costa Mesa on Saturday, September 7 and will include a live and silent auction. Duchenne is a progressive muscle-wasting disease that impacts one in 3,500 boys. Boys are usually diagnosed at age 5, are in a wheelchair by 12 and most dont live past their mid-20s. CureDuchenne … Continue reading →

(PRWEB) August 05, 2013 Please join the Peach State Quartet at Sharon Baptist Church, McDonough, Georgia on Saturday, September 7 at 5:00 p.m. for music and dinner to benefit the FSH Society, a world leader in combating muscular dystrophy. The Peach State Quartet, a male southern gospel group that has been singing together since 1972, will perform, followed by a Hawaiian-themed dinner. The ensemble was named one of the top 5 male quartets for 2012 by the Southeastern Southern Gospel Music Conference. Their single released to national radio, "Right on Time God," hit the Christian Voice Top 100 Chart in the first month it was released, and has climbed the chart every month since then. Were a group of guys that love to have fun while we are communicating the gospel of Jesus Christ through song, says the groups leader, baritone Doug Stroup. Opening for the Peach State Quartet will be the locally celebrated Fulmer Sisters, Nashville singer-songwriter Phil Bennett, and Virginia Wyckoff, the newly crowned National American Miss Georgia Teen 2013. Bennett was himself diagnosed with FSH muscular dystrophy, as was Wyckoffs sister Carden. Tickets are on sale now and proceeds from the event will go to the FSH … Continue reading →

Heading to high school is a challenge for any kid. There are new friends, new teachers and the normal ups and downs of being a teenager. For 15-year-old Chance Hawkins, the road is even tougher. Chance hasDuchennemuscular dystrophy, a genetic disease that causes muscle weakness and deterioration. Its often fatal by age 25. Still, Chance is an upbeat kid. He's one of the students were following from 8th grade through high school for the series Class of 17, part of KERAs American Graduate initiative. The KERA News story. Like a lot of teenage boys, Chance Hawkins is spending the summer watching his favorite TV shows, playing video games and hanging out with family. Twice a week, he also visits a pediatric rehab center for kids in his hometown of Fort Worth. Chance cant move his body much and gets around in a wheelchair. That's where physical therapist Nathan Morgan comes in. Morgan lays Chance down on an elevated cushioned mat. He stretches parts of the boy's body and applies pressure in some areas. Chance rests his head on a multicolored bean bag. The therapy helps him feel more comfortable. Its not all business, though. On a recent afternoon, they talk … Continue reading →