Category Archives: Muscular Dystrophy Treatment

Barry Byrne, M.D., Ph.D. University of Florida (USA) Michelle Eagle, Ph.D., M.Sc., MCSP Atom International Ltd (UK) Richard Finkel, M.D. Nemours Children's Hospital (USA) Pat Furlong Parent Project Muscular Dystrophy (USA) Kan Hor, M.D. Nationwide Children's Hospital (USA) John Jefferies, M.D. Cincinnati Children's Hospital Medical Center (USA) Oscar Henry Mayer, M.D. Children's Hospital of Philadelphia (USA) Eugenio Mercuri, M.D., Ph.D. Catholic University of the Sacred Heart (Italy) Francesco Muntoni, M.D. University College London (UK) Ron Victor, M.D. Cedars-Sinai Medical Center (USA) Thomas Voit, M.D. University College London (UK) Linda Marbn, Ph.D., president and CEO of Capricor, said, "We are gratified to have the attention and support of some of the foremost thought leaders in Duchenne muscular dystrophy, several of whom we have been working with since last fall. Following our recent announcement of positive clinical data in DMD with CAP-1002, we have formalized these relationships by assembling what we believe is a world-class advisory board of Duchenne specialists." "With their collaboration, we are planning the next steps in CAP-1002's development in DMD, including the design of a randomized, double-blind, placebo-controlled clinical trial of intravenous, repeat-dose CAP-1002 in which we expect to begin patient enrollment in the latter half of this … Continue reading →

What do expensive drugs have in common? A new study breaks it down. Boston Business Journal According to the study, four of the drugs in the top tier are sold by local companies: Duchenne muscular dystrophy treatment Exondys 51, made by Sarepta Therapeutics (Nasdaq: SRPT); spinal muscular atrophy drug Spinraza, made by Biogen (Nasdaq: BIIB); ... Read more here: What do expensive drugs have in common? A new study breaks it down. - Boston Business Journal … Continue reading →

What is Muscular Dystrophy? This disease will cause the muscle fibers in your body to weaken and begin to break down making your muscles susceptible to damage. Muscular Dystrophy is not just one disease but an umbrella term for a list of more than thirty muscle related diseases. This disease affects your skeletal or voluntary muscles. These are the muscles that control the movements of your arms, legs, and trunk. It can also affect involuntary muscles such as the ones in your stomach and even your heart muscles. It is a chronic disease that affects your mobility. This disease can start in infancy or childhood or it may wait to appear when a person reaches middle age. Muscular Dystrophy is a disease that is more prominent in boys than girls and is a progressive disease. There are more than fifty thousand people in the United States that are suffering from some type of Muscular Dystrophy. Muscular Dystrophy has nine types that are more commonly known. They are based on the age that the symptoms first appear and where the location is of the muscles that are involved. The type is also based on how fast or slow that the symptoms … Continue reading →

Timothy Stoklosa, a gifted artist who battled Duchenne muscular dystrophy, died on May 12. He was 33. Stoklosa worked as art director at The VIA Agency in Portland and was pursuing an art degree at the University of Southern Maine. He was an outspoken advocate for adults with disabilities, and worked with legislators to create laws giving him and others access to housing and direct support services. He was remembered by family and friends on Tuesday as a creative and inspiring guy who had a tremendous impact on others. He always rose to the occasion in spite of so many difficulties, said his mother, Karen Anderson, of Ocean Park. He never spent a day in bed feeling sorry for himself. He lived his life to the fullest on every level. Stoklosa grew up in Ocean Park and was diagnosed with Duchenne muscular dystrophy at age 3. At age 9, he lost his ability to walk and began using a wheelchair. Shortly after, he got his first electric-powered wheelchair. It was total freedom for him, his mother said. He would hit the road and go to Old Orchard Beach to buy pizza. He attended Thornton Academy and graduated in 2003. There, … Continue reading →

Weekly steroids strengthen, repair muscles Science Daily McNally initiated the research because she wanted to understand how prednisone -- which is given to treat individuals with a form of muscular dystrophy called Duchenne Muscular Dystrophy -- prolongs patients' ability to walk independently and stay out ... Read the original post: Weekly steroids strengthen, repair muscles - Science Daily … Continue reading →

May 16, 2017 Weekly doses of glucocorticoid steroids, such as prednisone, help speed recovery in muscle injuries, reports a new Northwestern Medicine study. The weekly steroids also repaired muscles damaged by muscular dystrophy. The studies were conducted in mice, with broad implications for humans. One of the major problems of using steroids such as prednisone is they cause muscle wasting and weakness when taken long term. This is a significant problem for people who take steroids for many chronic conditions, and can often result in patients having to stop steroid treatments. But the new study in mice showed weekly dosesrather than daily onespromote muscle repair. "We don't have human data yet, but these findings strongly suggest some alternative ways of giving a very commonly used drug in a manner that doesn't harm, but in fact helps muscle," said lead investigator Dr. Elizabeth McNally, the Elizabeth J. Ward Professor of Genetic Medicine at Northwestern University Feinberg School of Medicine and a Northwestern Medicine physician. McNally also is the director of the Center for Genetic Medicine at Feinberg. The study was published online in May in the Journal of Clinical Investigation. The study showed prednisone directs the production of annexins, proteins that … Continue reading →

May 16, 2017 01:02 ET | Source: Santhera Pharmaceuticals Holding Ltd multilang-release Liestal, Switzerland, May16, 2017 - Santhera Pharmaceuticals, a specialty pharmaceutical company focused on the development of innovative treatments for rare mitochondrial and neuromuscular diseases, announces that two posters with data from the positive phase III DELOS trial in patients with Duchenne muscular dystrophy (DMD) will be presented at the 113th American Thoracic Society (ATS) International Conference in Washington D.C. Throughout the conference, which runs May19-24, 2017, Santhera will host a clinical trial booth to inform clinicians on the new SIDEROS trial. The SIDEROS trial is a phase III, double-blind, randomized, placebo-controlled trial that evaluates the efficacy, safety and tolerability of idebenone in slowing the rate of respiratory function decline in DMD patients. Patients with reduced respiratory function on any stable glucocorticoid treatment regimen are eligible, regardless of their underlying dystrophin mutation or ambulatory status. More information is available on the new SIDEROS study website at http://www.siderosdmd.com. Santhera will also facilitate a non-CME symposium featuring an expert panel consisting of: Topics of the symposium include the pathophysiology of DMD and data around the importance of preserving respiratory function in DMD. "Santhera is pleased to have the opportunity to showcase … Continue reading →

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Solid Biosciences announced today that new data from two preclinical studies reinforce the potential of its investigational microdystrophin gene therapy, SGT-001, to be an effective treatment approach for Duchenne muscular dystrophy (DMD). The preclinical data, which were presented at the American Society of Gene and Cell Therapy (ASGCT) 20th Annual Meeting, demonstrated that a single administration of SGT-001 resulted in sustained and significant microdystrophin expression and improvements in muscle function, with no observed immune response. The Company plans to initiate clinical trials for SGT-001 in the second half of 2017. These preclinical data show the potential efficacy, durability and tolerability of SGT-001, and give us further confidence in our plan to initiate our clinical program later this year, said Joel Schneider, Ph.D., vice president of Research and Development at Solid Biosciences. I would like to thank our academic partners for their work to help us build one of the most robust gene therapy preclinical datasets in DMD and for sharing our mission to bring meaningful treatments to patients with this devastating disease. Data from the studies were presented by Solids collaborators at the University of Missouri and Texas A&M University in two posters: In both studies, administration of … Continue reading →

Bradley County Fire Department kicks off "Fill the Boot" for muscular dystrophy (Photo: WTVC) The Bradley County Fire Department is kicking off it's annual Fill the Boot campaign for the Muscular Dystrophy Association to help kids and adults suffering from muscular dystrophy, ALS and other related muscle-debilitating diseases. Their goal is to help them live longer and grow stronger. More than 65 firefighters from the BCFD will hit the streets and storefronts with boots in hand to ask for donations on May 11th, 12th, and 13th from 9 am to 9 pm. You can find firefighters at the Walmart on McGrady Dr in Cleveland, the Walmart on Paul Huff Pkwy in Cleveland, and at Station 9 off Hwy 11 in Charleston, TN. Contributions from the campaign help fund research and life-enhancing programs such as state-of-the-art support groups and Care Centers, including the MDA Care Center at Vanderbilt University Medical Center in Nashville. They also help send more than 80 local kids to the best week of the year at MDA summer camp at Camp Widjiwagan in Nashville. The summer camp is no cost to their families. We are thrilled to be working with the Bradley County Fire Department for another … Continue reading →

MEMPHIS, Tenn.--(BUSINESS WIRE)-- GTx, Inc. (GTXI) today announced the publication in the journal Human Molecular Genetics of results from preclinical studies supporting the potential efficacy of the Companys SARMs, and in particular GTx-026, for Duchenne Muscular Dystrophy (DMD) treatment. DMD is a rare genetic disorder characterized by progressive muscle degeneration and weakness, affecting approximately 18,000 boys in the United States. Studies examined the treatment of castrated wildtype mice, x-linked muscular dystrophy (mdx) mice, and dystrophin and utrophin double mutant (mdx-dm) mice with GTx-026. Other SARMs in the Companys portfolio, GTx-024 (enobosarm) and GTx-027, showed similar positive effects on muscle mass, function, and histological characteristics. DMD typically afflicts boys around three to five years of age followed by declining physical functions before attaining puberty. Current treatment options for DMD rely on corticosteroids to reduce inflammation, but unfortunately the prolonged use of corticosteroids results in hyperglycemia, osteoporosis, and muscle wasting, which are all counterproductive in this disease, said Ramesh Narayanan, Ph.D., Director, Center for Cancer Drug Discovery and Associate Professor, Department of Medicine, University of Tennessee and a consultant for GTx, Inc. We hypothesize that an androgen receptor agonist may reverse musculoskeletal complications and extend survival in these boys, and in preclinical … Continue reading →