Category Archives: Muscular Dystrophy Treatment

CAMBRIDGE, Mass., Sept. 10, 2012 /PRNewswire/ --DART Therapeutics, LLC, an innovative, new-model biotechnology firm focused on developing therapies for Duchenne Muscular Dystrophy (DMD), announced today the appointment of its executive team. Co-Founder and Executive Chairman Eugene W. Williams transitions to a full-time role as CEO, and Ernest D. Bush Ph.D. joins as Chief Scientific Officer. DART also adds Dr. Elliot Goldstein as Chief Medical Officer and Cabot Brown as Chief Financial Officer, both on a part-time basis. Each will apply more than 25 years of executive-level experience in his respective field to expedite development of a therapy for DMD. DART was co-founded in 2010 by two leading patient foundations in DMD, Charley's Fund and the Nash Avery Foundation, along with Executive Chairman Gene Williams. It has derived funding to date exclusively from these foundations. As a patient-funded biotechnology firm, DART reflects a new model for research and drug development centered on the needs of families affected by DMD and potentially transportable to other pediatric rare diseases. While DART is patient-funded and founded, it benefits from professional staff and business processes like any other well-managed biotech. In its first two years, DART acquired a promising clinical stage anti-fibrotic drug candidate and … Continue reading →

There's a hard, urgent logic to Jenn Mcnary's plea: Her 13-year-old son, Austin, is wasting away from an incurable genetic disease, while her 10-year-old with the same disorder has been helped by testing a cutting-edge drug that's not yet approved for sale. "I would love to get him on this medication to give him as good a chance as Max has gotten," the Vermont mom says. "At this stage anything that can stop the progression is a lifesaver." There's an equally compelling logic to the strategy of Sarepta Therapeutics CEO Chris Garabedian, who recently reported clinical-trial results that suggest the drug may be the first effective treatment for slowing Duchenne muscular dystrophy. "We could spend all our time answering phone calls from parents who would like our drug, and putting in the paperwork to the FDA" for individual "compassionate use" cases in which Sarepta can't charge money, he says. "That is the surest path to insolvency." Sarepta -- until recently called AVI BioPharma and based in Bothell but now headquartered outside Boston -- may be one of those biotech fairy tales that sustains belief in the entire industry: A little company that brings a novel lifesaving drug to market. But … Continue reading →

Sarepta Therapeutics (Nasdaq: SRPT) announced an at-the-market equity offering sales agreement with Citadel Securities on Tuesday. Under the terms of the agreement, the company can offer and sell up to $40 million of its common stock. With Sarepta selling (or at least clearing the way to sell), is now a good time for investors to buy? Let's take a look. Buy? The single most compelling reason to buy Sarepta is the promise of its eteplirsen drug in treating Duchenne muscular dystrophy, or DMD. There are currently no disease-modifying therapies available for the condition. Eteplirsen shows great potential for treating DMD. Sarepta announced positive results in April from its phase 2b clinical trial. The study found that the drug produced consistent levels of dystrophin, the protein needed for muscular function that is lacking in patients with DMD. In July, the company announced even better news. Treatment with eteplirsen over a 36-week period achieved significant clinical benefits for patients. Dr. Jerry Mendell, director of the centers for gene therapy and muscular dystrophy at Nationwide Children's Hospital and principle investigator of the phase 2b study, stated that eteplirsen was "the most promising advance to treat the underlying cause of muscular dystrophy I've seen … Continue reading →

Firefighters Fill-the-Boot for muscular dystrophy The Grand Junction Fire Department kicked of its annual Fill-the-Boot campaign, Saturday. Its part of an international effort to raise money for muscular dystrophy, a disease affecting hundreds of families in Western Colorado. "More than 40 diseases make up muscular dystrophy and there is no cure yet," Kendall Montagriff, with the Muscular Dystrophy Association, said. Children with muscular dystrophy face difficulty everyday because of the disease. 9-year-old Shane Walz is no exception. Back in 2005 he was diagnosed with an unknown type of MD. During Shanes uphill battle, the Muscular Dystrophy Association has helped pay for his testing, wheelchairs, and other equipment. His grandmother, Danna Michels, says "for the family, what MDA kicks in is money towards research and trying to find a cure." Shane has been working with Children's Hospital in Denver, trying to find a more specific diagnosis in hopes of getting successful treatment. View original post here: Firefighters Fill-the-Boot for muscular dystrophy … Continue reading →

ScienceDaily (Aug. 30, 2012) Scientists have moved closer toward helping people grow big, strong muscles without needing to hit the weight room. Australian researchers have found that by blocking the function of a protein called Grb10 while mice were in the womb, they were considerably stronger and more muscular than their normal counterparts. This discovery appears in the September 2012 issue of The FASEB Journal. Outside of aesthetics, this study has important implications for a wide range of conditions that are worsened by, or cause muscle wasting, such as injury, muscular dystrophy, Type 2 diabetes, and problems produced by muscle inflammation. "By identifying a novel mechanism regulating muscle development, our work has revealed potential new strategies to increase muscle mass," said Lowenna J. Holt, Ph.D., a study author from the Diabetes and Obesity Research Program at the Garvan Institute of Medical Research in Sydney, Australia. "Ultimately, this might improve treatment of muscle wasting conditions, as well as metabolic disorders such as Type 2 diabetes." To make this discovery, Holt and colleagues compared two groups of mice. Once group had disruption of the Grb10 gene, and were very muscular. The other group, where the Grb10 gene was functional, had normal muscles. … Continue reading →

EDMOND At age 6, when most kids are playing with their friends or getting scolded by their parents, Amy Jenkins was diagnosed with Charcot-Marie-Tooth disease. Named for the three physicians who first identified it in 1886, Charcot-Marie-Tooth disease is caused by mutations that affect the normal function of the peripheral nerves. The degeneration of motor nerves causes muscle weakness and atrophy in the extremities (arms, legs, hands or feet). In some cases the degeneration of sensory nerves results in a reduced ability to feel heat, cold and pain. Wednesday afternoon, as individuals, organizations and businesses are raising funds for the Muscular Dystrophy Association, Jenkins was at Edmonds Fire Station 1, located next to the University of Central Oklahoma campus, to talk about her story. Jenkins said several relatives including her father have the same disease, which was the closest fit to their disorder. Genetic testing confirmed a difference. She said a cure for her disease hasnt been found yet, but MDA-funded research is making a difference. Try and imagine as an individual paying for six years of research at $86 a minute, Jenkins said. Thats not feasible. So having that is hope toward finding a treatment, toward finding assistance and … Continue reading →

GREEN BAY - When you live with a disease like muscular dystrophy every day can bring new challenge. Patients in Northeast Wisconsin have a place to go for regular check-ups on certain days. It's called Clinic Day. One of the doctors in the area who helps care for patients is Dr. Terence Edgar with Prevea Clinic in Green Bay. Edgar says a team of medical professionals come together to bring different skills directly to the patients. They monitor everything from heart to lungs, all the way to the patients body functions. Edgar says treatment has come a long way in the last ten years. So the advances we have now, now we don't just look at can we cure - but how can we make your heart function the best and so there is a multitude of different strategies that we use, said Dr. Edgar. MDA raises money to support treatment and research to find cures for some 40 neuromuscular diseases. Be sure to join us for the MDA Show of Strength on Sunday night from 7 until 10. More: Clinic Day for muscular dystrophy … Continue reading →

Fill the Boot or MDA Lock-Up are events that may ring a bell for some. You might have helped to collect or donate to the Muscular Dystrophy Association's well-known fundraisers. Thanks to recently raised funds, a local clinic could open by the end of fall. "We have such great support from the community. Fill the Boot generated about $66,000 and $5,000 of that was donated from El Paso Electric to kick that campaign off. Then we did just finish with the executive Lock-Up over at Landry's, which will bring in close to $60,000. Of all that money, 77 cents of every dollar does stays local," said Kathleen Born, executive director of MDA. Every year the El Paso Fire Department teams up with MDA for the Fill the Boot campaign in which firefighters hit the streets and collect donations from drivers. Restaurants also help the nonprofit by hosting various MDA Lock-Ups. Local leaders are nominated to be locked up in MDA's jail and have to raise bail through donations. These events help the nonprofit provide counseling and treatment for more than 400 people throughout El Paso, Carlsbad, Roswell and Artisan who have MD. "There's 43 different neuromuscular diseases, including ALS, and … Continue reading →

ScienceDaily (Aug. 27, 2012) Muscular dystrophy is a complicated set of genetic diseases in which genetic mutations affect the various proteins that contribute to a complex that is required for a structural bridge between muscle cells and the extracellular matrix (ECM) that provides the physical and chemical environment required for their development and function. The affects of these genetic mutations in patients vary widely, even when the same gene is affected. In order to develop treatments for this disease, it is important to have an animal model that accurately reflects the course of the disease in humans. In this issue of the Journal of Clinical Investigation, researchers at the University of Iowa report the development of a mouse model of Fukuyama's muscular dystrophy that copies the pathology seen in the human form of the disease. By removing the gene fukutin from mouse embryos at various points during development, researchers led by Kevin Campbell were able to determine that fukutin disrupts important modifications of dystrophin that prevent the muscle cells from attaching to the ECM. Disruption of the gene earlier in development led to a more severe form of the disease, suggesting that fukutin is important for muscle maturation. Disruptions in … Continue reading →

by Jenne Lajiun. Posted on August 25, 2012, Saturday Tham and her son, Edmund, at the event. Tham is climbing Mount Kinabalu for the first time to help raise awareness on Duchnenne. KOTA KINABALU: Parents with children afflicted by Duchenne muscular dystrophy may only have one simple wish that treatment would enable their children to outlive them. But sadly, this disease has stoppably robbed them of their beloved children too early during their lifetime, more often than not in their teens. Tham Chew Yen, 28, shared her wish for treatment to be made available to her six years old son, Edmund Wong, who was diagnosed with the disease when he was two years old. Having experienced the loss of her eldest brother to a similar condition 19 years ago, she knows all too well the pain one has to endure in the face of such adversity. I wish for a miracle and a cure. I saw what Duchenne did to my brother and it was very difficult to accept his passing because we were so close, Tham said. He died when he was 13 years old. We were very close and so when he died, I felt a knife thrusted … Continue reading →