Category Archives: Muscular Dystrophy Treatment

Hilo native Daryl Sammy Sampaga is at it again. A year after making headlines in early 2014 for trekking the Big Island-round to raise awareness of muscular dystrophy and to support keiki with disabilities, Sampaga will take on the island of Kauai by two means: walking and paddling. Sampaga will begin the journey, nicknamed Operation Paddle and Walk for Dreams Kauai Island 2015, on Jan. 17. Before I started (the Big Island) walk, I had mentioned to everyone that I was going to hit this island and then hit all the other islands, Sampaga said. They doubted me the first time, saying you not going make em, and no one every tried and I said I will try, even if I got to crawl, I will crawl and I did. The Kauai project, the latest of his nonprofit, Operation Sammy Muscular Dystrophy, will take Sampaga, who has muscular dystrophy, which affects his leg muscles, on a more than 100-mile trip around the Garden Island. The journey will comprise 78 miles of walking and up to 32 miles of paddling along the Na Pali Coast, Sampaga told West Hawaii Today on Tuesday. He will begin the feat first by paddling the … Continue reading →

Dear Penticton and surrounding communities: 2014 has been another successful year thanks to the massive amount of support we received from the people we serve. With your continued support, The Penticton Professional Firefighters Charitable Society raised and donated another $41,000 to local, provincial and national charities. A few of the charities/programs we donated to are: BC Burn Fund, Muscular Dystrophy Canada, Miss Penticton, South Okanagan Women in Needs Society, Penticton Regional Hospital Pediatrics Ward, Penticton Minor Hockey, The Penticton Safety Village, Have a Heart Telethon, OSNS Telethon, and Toys for Tots to Teens. We would like to thank everyone who came out to our events and supported our causes; as well we are blessed to have made some great partnerships within our own community with businesses who have become corporate sponsors and partners at some of our events. Britco has helped us in many ways like constructing the platform deck for our rooftop campouts and donating money towards our fundraising. Scotia Bank has become great partners at a few of our events like the Peach City Beach Cruise Pancake Breakfast and The Muscular Dystrophy Rooftop Campout, and has matched our fundraising dollar for dollar at these events. Safeway has partnered … Continue reading →

Donors Help to Anupoju Sivakumar for Muscular Dystrophy Treatment Bathuku Bandi - Anupoju Sivakumar Asking for Help to Muscular Dystrophy Treatment VANITHA TV - First Women Centric Channel in India Click here to Subscribe f... By: Vanitha Tv … Continue reading →

Dont underestimate a fathers determination. Ty Ballou of Ben Avon was eager for a new treatment for his son Ryan, now 27, who has Duchenne muscular dystrophy, uses a wheelchair and already has outlived what his life expectancy would have been. After helping to inspire a researcher to test a new drug regimen, he raised nearly $250,000 over six years to fund research at the Ohio State University Wexner Medical Center. He collected contributions to complete marathons and other distance races, raised money from the sale of Ballou Skies peanut butter for two years at Giant Eagle and continues operating the web site http://www.ballouskies.com to seek donations and provide information about the disease that predominantly affects males. The results are in. A study published today in The Lancet Neurology and led by Subha Raman, a cardiologist and professor at the medical center, shows that a combination of prescription drugs was used successfully to reduce the progression of heart failure in those with Duchenne. Those results follow Mr. Ballous sons success with a similar regimen of drugs hes been taking for seven years. Those include the ACE inhibitor lisinopril and the diuretic spironolactone, which typically are used to treat advanced heart … Continue reading →

Signs of Duchenne muscular dystrophy (DMD) can start to appear in boys as young as six, leading to deterioration of the heart muscles and ultimately death. Pharmaceuticals aimed at controlling high blood pressure have been used to treat the one in 3,500 young males suffering from the condition, but a new study suggests that a novel combination of these drugs could slow the decline in heart function earlier on, and in promising new ways. Led by cardiologist Dr Subha Raman, a professor at Ohio State University Wexner Medical Center, a research team explored the effects of combining heart failure drugs to treat heart muscle disease over a twelve month period. A group of 42 boys suffering from DMD and heart muscle degeneration were treated with either an ACE inhibitor or an angiotensin receptor blocker (ARB), drugs used to treat elevated blood pressure. Among the 42 boys, a random group was also treated with eplerenone, another blood pressure medication, while the rest were given a placebo. The subjects underwent cardiac MRIs before, halfway through and then following the 12 month study period, enabling the researchers to track the condition of their cardiovascular health. The researchers reported that the patients receiving eplerenone … Continue reading →

Research finds early detection, combination of drugs protects hearts of boys longer VIDEO:In a new study led by Dr. Subha Raman of The Ohio State University Wexner Medical Center, researchers were able to dramatically slow the rate of heart damage in patients... view more COLUMBUS, Ohio - Early use of available heart failure drugs slows the progressive decline in heart function before symptoms are apparent in boys and young men with Duchenne muscular dystrophy (DMD), according to a new study published online by The Lancet Neurology. Dr. Subha Raman, a cardiologist and professor at The Ohio State University Wexner Medical Center, led a team of DMD experts at multiple sites in a clinical trial that tested the combination of eplerenone and either an ACE inhibitor or an angiotensin receptor blocker (ARB) to decrease the progression of heart muscle disease, a leading cause of death in boys and young men with DMD. "We believe this research offers evidence that supports the early use of these readily available medications," said Raman, who is also the lead author of the study. Dr. Linda Cripe, a pediatric cardiologist and co-investigator at Nationwide Children's Hospital in Columbus, added, "This could quickly become standard of care … Continue reading →

Early use of available heart failure drugs slows the progressive decline in heart function before symptoms are apparent in boys and young men with Duchenne muscular dystrophy (DMD), according to a new study published online by The Lancet Neurology. Dr. Subha Raman, a cardiologist and professor at The Ohio State University Wexner Medical Center, led a team of DMD experts at multiple sites in a clinical trial that tested the combination of eplerenone and either an ACE inhibitor or an angiotensin receptor blocker (ARB) to decrease the progression of heart muscle disease, a leading cause of death in boys and young men with DMD. "We believe this research offers evidence that supports the early use of these readily available medications," said Raman, who is also the lead author of the study. Dr. Linda Cripe, a pediatric cardiologist and co-investigator at Nationwide Children's Hospital in Columbus, added, "This could quickly become standard of care for patients with DMD." Raman and her team based this trial on their earlier lab findings that showed this combination of medicines reduced muscle damage and preserved function in an animal model of DMD. In this trial, researchers enrolled 42 boys with DMD who also showed evidence … Continue reading →

Although drugs are being developed to try and control genetic diseases, more focus should be on prevention, experts say. Since diseases like muscular dystrophy and Huntingtons Disease are passed down through generations, it is possible to prevent their spread, according to S.H. Subramony, professor of neurology and paediatrics at the McKnight Brain Institute at University of Florida. He was delivering the Neurokrish Lecture during the fourth anniversary celebrations of TRIMED, a Chennai-based healthcare start-up focussed on chronic diseases, on Saturday. Most people may not develop a genetic disease till they are 40 or 50, by which time they would have already had children. Instead, if people who have a history of genetic diseases in their family are tested, they can make an informed choice of whether to have children or not, says Dr. Subramony. Ennapadam S. Krishnamoorthy, founder and director of TRIMED, says they are working towards integrating modern medicine with traditional systems like Ayurveda and yoga. We are looking at tying up with doctors and working together to provide overall treatment, he says. An exhibition of work by and of people with disabilities was launched on the occasion. The exhibition will be on at the four TRIMED centres in … Continue reading →

From left to right: Stanley Nelson, MD, Center for DMD at UCLA; Thomas Sugar, PhD, Arizona State University Polytechnic School; Tom Egan, SRI International; Roy Kornbluh, SRI International; Laura Case, DPT, Duke University School of Medicine; Keith Van Houten; Annie Kennedy, PPMD; Matt Arnold, Solid Ventures; Lee Sweeney, PhD, University of Pennsylvania; Tina Duong, DPT, Children's National Medical Center; Annie Ganot; Ilan Ganot, Solid Ventures; Eytani Ganot; Andrey Zarur, PhD, Solid Ventures; Brenda Wong, MD, Comprehensive Neuromuscular ... Parent Project Muscular Dystrophy logo. (PRNewsFoto/Parent Project Muscular Dystrophy) HACKENSACK, N.J., Dec. 18, 2014 /PRNewswire-USNewswire/ --Parent Project Muscular Dystrophy (PPMD), a nonprofit organization dedicated to the fight to end Duchenne muscular dystrophy (Duchenne), announced today a collaboration with Solid Ventures (Solid) to engage SRI Internationalin the research and development of the "Solid Suit," a soft, wearable assistive device for people with Duchenne. Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 3,500 to 5,000 live male births. The loss of a key muscle protein called dystrophin causes muscle wasting and weakness, eventually leading to the loss of ambulation, difficulty breathing, and heart failure. Death typically occurs in the mid-to late 20s. The Solid … Continue reading →

PBR Staff Writer Published 18 December 2014 Pfizer has started patient enrollment in a multicenter Phase II clinical trial of PF-06252616, an experimental, infused, anti-myostatin monoclonal antibody, to treat Duchenne muscular dystrophy (DMD). The trial is designed to evaluate the safety, tolerability and efficacy of PF-06252616 in boys aged six to less than ten years old diagnosed with DMD regardless of genotype. DMD is genetic disorder characterized by progressive muscle degeneration and weakness. Myostatin is a naturally occurring protein that helps control muscle growth and it is believed that blocking its activity may have potential therapeutic application in treating muscle wasting diseases such as DMD. Pfizer Rare Disease Research Unit senior vice-president and chief scientific officer Kevin Lee said: "DMD is a devastating and debilitating disease impacting approximately 1 in 3,500 male births worldwidei with no current treatment options. "We are pleased to be taking this important next step in the development of PF-06252616 as an investigational therapy for DMD in the hopes of potentially bringing a much-needed therapy to individuals and families with this devastating disease." Based on the proposed mechanism of action of PF-06252616, the company is exploring whether there is the potential to increase muscle mass and … Continue reading →