Category Archives: Muscular Dystrophy Treatment

Public release date: 26-Jun-2012 [ | E-mail | Share ] Contact: Nick Miller nicholas.miller@cchmc.org 513-803-6035 Cincinnati Children's Hospital Medical Center CINCINNATI The first landmark randomized clinical trial for a cardiac drug regimen in Duchenne muscular dystrophy (DMD) is testing whether earlier treatment can stop or slow down heart damage that usually kills people with the disease. The study is a collaboration of Cincinnati Children's Hospital Medical Center, Ohio State University (OSU) and The Christ Hospital in Cincinnati. Extensive research including studies in mouse models of DMD suggests an anti-fibrosis drug long used to treat heart attack victims, eplerenone, could help people with the disease, said Kan Hor, MD, a principal investigator and a physician/researcher at Cincinnati Children's. Disease-associated heart damage is the leading cause of death in patients between ages 20 and 30 the maximum life span for people with DMD. A key element of the new treatment approach is using enhanced cardiac magnetic resonance imaging, Hor said. The technique more sensitive than standard echocardiogram tests now used in DMD is designed to catch minute declines in heart function much earlier in a child's life. "Standard diagnostic techniques rarely catch significant heart damage in DMD in the first decade, although … Continue reading →

Public release date: 21-Jun-2012 [ | E-mail | Share ] Contact: Mike Wolterbeek mwolterbeek@unr.edu University of Nevada, Reno RENO, Nev. Leading muscular dystrophy researcher Dean Burkin, of the University of Nevada School of Medicine summarizes the impact of a new protein therapeutic, MG53, for the treatment of Duchenne muscular dystrophy in an article published this week in Science Translational Medicine. "This is a focus article in which we summarize the impact of MG53 protein therapy as a treatment option and discuss the increasing number of new protein therapeutics being developed for the muscular dystrophies, including laminin-111 developed in our laboratory," Burkin, a pharmacological researcher and associate professor, said. The article, "A Molecular Bandage for Diseased Muscle," co-authored by Ryan Wuebbles, a post-doctoral student in Burkin's lab in the University's Center for Molecular Medicine, is a review of the current status of therapeutic developments in the muscular dystrophy research field. These therapies represent significant strides and show great promise in treatment of muscular dystrophy. Approaches to treat Duchenne muscular dystrophy include gene replacement therapy, gene repair and myoblast cell transfer. In a study on MG53 by Noah Weislander and colleagues, presented in the same issue of Science Translational Medicine, it was … Continue reading →

Jun 22 2012 by Clair Fullarton, Kilmarnock Standard A TEAM of fundraisers are using pedal power to raise cash for a little boy with Duchenne Muscular Dystrophy. And the plucky cyclists were recently given the backing of former Miss Scotland Jennifer Reoch. Led by Dr Chris Govender, whose little boy Brandon is the inspiration behind the marathon cycle, the cyclists will travel from Scotlands most southerly point, the Mull of Galloway, to John OGroats. This 450-mile trek will kick off on Friday, July 6, and is expected to take the four-man team eight gruelling days to complete. Those taking part are David Cooke (Prestwick), Lenny Anderson (Howwood), Chris Govender (Kilmarnock) all staff at Dykebar Hospital, Paisley, Scotland, and Aaron Bayne, Brandons cousin who is in fourth year at Kilmarnock Academy. Kilmarnock man Chris said: We will be accommodated by local fire stations en-route and our nightly stops include Girvan, Kilmarnock, Arrochar, Killin, Newtonmore, Inverness, Helmsdale reaching John O Groats on Friday, July 13. Anybody interested in joining us for any part of our trip is most welcome! Gerry, owner of Ayrshire Cycle Services (ACS) in Prestwick, has sponsored the team with three bicycles and cycling equipment for the journey. Chris … Continue reading →

ANN ARBOR, MI--(Marketwire -06/21/12)- Phrixus Pharmaceuticals, Inc., a clinical-stage, specialty pharmaceutical company focused on innovative therapies for Duchenne muscular dystrophy (DMD) and heart failure, today announced that it has received an SBIR Phase 1 award for $623,000 from the National Institutes of Health (NIH) for its grant application titled "Treatment of Muscular Dystrophy-Associated Dilated Cardiomyopathy with P-188." "This funding constitutes validation for the potential utility of Carmeseal in DMD. We expect that it will allow us to extend administration of Carmeseal to a new, clinically relevant route of administration, subcutaneous delivery," said Thomas A. Collet, president and CEO. "Completion of the work funded by this grant will significantly advance our knowledge of how to best turn Carmeseal into a chronic therapy suitable for daily administration," adds Dr. Bruce Markham, Vice President of Research and Chief Scientific Officer. DMD is the most devastating of the muscular dystrophies. No drug is approved for its treatment. It is a genetic disease that affects about one out of every 3,500 boys. Approximately 20,000 boys and young men live with this disease in the United States. The hallmarks of DMD are skeletal muscle weakness, respiratory distress, and cardiomyopathy. It is a degenerative disease that leads … Continue reading →

Newswise Nationwide Childrens Hospital CEO Steve Allen, MD, will serve as co-chair of the first Transforming Duchenne Care workshop to be held June 27 and 28, 2012, in Ft. Lauderdale, FL. The national symposium, held by Parent Project Muscular Dystrophy, will gather leadership from top medical institutions, neuromuscular experts and patient representatives with the ultimate goal of establishing a network of centers of excellence for the care and treatment of children with Duchenne muscular dystrophy. In addition to Dr. Allen, Nationwide Childrens will be represented by Jerry Mendell, MD; Kevin Flanigan, MD; and Linda H. Cripe, MDthree prominent pediatric specialists in the Neuromuscular Disorders program. Dr. Mendell is an attending neurologist, Director of the Center for Gene Therapy at The Research Institute at Nationwide Childrens and Director of the Paul D. Wellstone Muscular Dystrophy Cooperative Research Center. He is also Co-director of the Muscular Dystrophy Association Clinic at Nationwide Childrens and serves as Professor of Pediatrics, Neurology, Pathology, and Physiology and Cell Biology at The Ohio State University College of Medicine. Dr. Flanigan, also a neurologist at Nationwide Childrens, is a principal investigator in the Center for Gene Therapy in The Research Institute and Professor of Pediatrics at The Ohio … Continue reading →

ScienceDaily (June 20, 2012) Throughout the lifecycle, injury to the body's cells occurs naturally, as well as through trauma. Cells have the ability to repair and regenerate themselves, but a defect in the repair process can lead to cardiovascular, neurological, muscular or pulmonary diseases. Recent discoveries of key genes that control cell repair have advanced the often painstaking search for ways to enhance the repair process. A new study by researchers from the University of Medicine and Dentistry of New Jersey (UMDNJ)-Robert Wood Johnson Medical School reports that the protein MG53, previously shown to be the key initiator in the cell membrane repair process, has the potential to be used directly as a therapeutic approach to treating traumatic tissue damage. "We studied the use of MG53 in treating muscular dystrophy by targeting the protein directly to the damaged muscle. The direct application of MG53 slowed the development of the disease by repairing damaged muscle membranes," said Noah Weisleder, PhD, assistant professor of physiology and biophysics and corresponding author of the study. "Our findings also suggest that MG53 could be used in regenerative medicine to treat other human diseases in which traumatic cell injury occurs." The study established methods to produce … Continue reading →

Information contained on this page is provided by companies via press release distributed through PR Newswire, an independent third-party content provider. PR Newswire, WorldNow and this Station make no warranties or representations in connection therewith. SOURCE Parent Project Muscular Dystrophy HACKENSACK, N.J., June 20, 2012 /PRNewswire-USNewswire/ -- AVI BioPharma is teaming up with Parent Project Muscular Dystrophy (PPMD)'s endurance program, Run For Our Sons, to participate in the Rock 'n' Roll Seattle Marathon & Half Marathon on June 23, 2012. The goal is to raise money and awareness to help end Duchenne muscular dystrophy (Duchenne), the most common form of muscular dystrophy. (Logo: http://photos.prnewswire.com/prnh/20100119/DC39975LOGO) AVI and PPMD have a long history of working together to develop treatments that may help to prolong the lives of those living with Duchenne. "PPMD has always been grateful to the dedication AVI has shown to the Duchenne community as they continue to work on treatments that will help our sons live longer, stronger lives," said Pat Furlong, PPMD's President & Founder. "Now they are taking that dedication a step further by lacing up their running shoes and pounding the streets of Seattle to help raise awareness and end Duchenne." Participating alongside the AVI team … Continue reading →

Letter Signed by Nearly 120 Organizations, Latest PDUFA-Oriented Action HACKENSACK, N.J., June 13, 2012 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD), the nation's largest Duchenne muscular dystrophy (Duchenne) advocacy organization co-led a letter to key lawmakers that was signed by 119 organizations and that urges Congress to include the strongest patient engagement and rare disease provisions within the final use fee legislation. (Logo:http://photos.prnewswire.com/prnh/20100119/DC39975LOGO) Developed in partnership with the EveryLife Foundation for Rare Diseases and Genetic Alliance, the letter represents the latest of several actions PPMD has engaged in for months as Congress has developed the user fee package. Other groups signing onto the letter include the National Down Syndrome Society, the Muscular Dystrophy Association, the American Association on Health and Disability, the National Tourette Syndrome Association, and Special Olympics. The House and Senate both passed user fee bills that included provisions aimed at accelerating Food and Drug Administration (FDA) review of therapies to treat rare diseases and to enable FDA to more aggressively engage external experts in reviewing candidate therapies. "The House and Senate should both be commended for including a number of provisions intended to accelerate the development of therapies for patients suffering from rare and life-threatening disorders like … Continue reading →

ABINGDON, UNITED KINGDOM--(Marketwire -06/12/12)- SUMMIT ACHIEVES SMT C1100 PHASE 1 DOSING MILESTONE THAT TRIGGERS MILESTONE PAYMENT Summit (SUMM.L), a UK drug discovery company, today announced that it has successfully passed a milestone in the Phase 1 trial of SMT C1100 for the treatment of the fatal genetic disease Duchenne Muscular Dystrophy ('DMD'), which triggered the final payment from a $1.5 million funding agreement with US-based DMD organisations. SMT C1100, an oral small molecule compound, is a potential disease-modifying drug that works by increasing, or upregulating, the amount of a naturally occurring protein called utrophin. The Phase 1 dose-escalation study in healthy volunteers was initiated in May 2012 and will now progress to the stage where participants receive multiple doses. Results from the trial are expected by the end of this year. The Phase 1 trial is being supported by a group of US-based DMD organisations: the Muscular Dystrophy Association, Charley's Fund, Cure Duchenne, the Foundation to Eradicate Duchenne, Nash Avery Foundation and Parent Project Muscular Dystrophy. "We are grateful for the continuing support from the DMD organisations as we make significant progress in the Phase 1 trial of SMT C1100," said Glyn Edwards, Chief Executive Officer of Summit. "The funding … Continue reading →

By Caroline Cheetham PUBLISHED: 17:17 EST, 11 June 2012 | UPDATED: 17:32 EST, 11 June 2012 As she watched her son Jack buzzing about at playgroup, Alex Johnson noticed the 18-month-old didnt seem as advanced physically as the other children. He couldnt jump as high, or run as fast, and he struggled to hold himself up when he was on the slide, says Alex. There wasnt anything more specific than that but to my eye it seemed noticeable. On Duchenne muscular dystrophy: 'I kept looking at the information and realised Jack wasn't going to get better. In fact, he was going to get worse,' said Alex Johnson, pictured with son Jack and husband Andy Worried, Alex took Jack to the local GP surgery on three occasions. However, the doctors were not concerned. And Alex was simply advised children develop at different stages and all catch up eventually, so she felt reassured. The GPs words had particular resonance for 27-year-old Alex and her husband Andy. Alex had nearly miscarried four times during her pregnancy, so when Jack was born in January 2008 safe and well, theyd said a silent prayer of thanks for their miracle. See original here: Duchenne muscular dystrophy: … Continue reading →