Category Archives: Muscular Dystrophy Treatment

As the editorial editor of his school newspaper and an active volunteer in causes ranging from Muscular Dystrophy Association to Junior Achievement, Mark Liston had hoped to become a grade school teacher when he graduated from West High School in 1972. Life got in the way. My dad died when I was 14. I was married at 18, and I had three kids by the time I was 22, said Liston, one of 54 students chosen by the Register Star as Young Americans in 1972. I started going to Rock Valley College. I liked school, and I wanted to finish, but I had to work. For many, the value of a college degree cannot be overstated. Exact estimates vary, but a person with a college degree earns about $1 million more on average over the course of a lifetime than someone with just a high school diploma. But Liston is one of the exceptions to the rule. After working in finance for seven years, Liston began working for a water treatment franchisor in 1981 because of his link to LTM Water Treatment now in Machesney Park. He liked the franchising industry and stayed with it, working with several more companies … Continue reading →

BURLINGTON, Mass.--(BUSINESS WIRE)-- Decision Resources, one of the worlds leading research and advisory firms focusing on pharmaceutical and healthcare issues, finds that interviewed experts unanimously identify a disease-modifying therapy as the highest unmet need in the treatment of Duchenne muscular dystrophy (DMD). Duchenne is the most common form of muscular dystrophy in children. Glucocorticoid treatment is the only available therapy for the treatment of DMD, said Decision Resources Analyst Richa Mukherjee, M.Sc., M.Phil., Ph.D. Its a symptomatic therapy that does not target the cause of the disease and its use is often accompanied by concerning side effects. The lack of a disease-modifying therapy to treat DMD presents a high unmet need for drug developers. According to Niche Markets and Rare Diseases: Muscular Dystrophy, Decision Resources forecasts the launch of four new agents to treat DMD by 2021. These therapies include GlaxoSmithKline/Prosensas GSK-2402968/PRO-051/drisapersen, Santhera/Takeda's Catena/Sovirmas idebenone, AVI BioPharmas eteplirsen/AVI-4658 and Prosensas PRO-044. Of these therapies, GSK-2402968, eteplirsen and PRO-044 are disease-modifying therapies that will heavily saturate their target patient segments however, given the small size of these segments, the market will still be very receptive to novel disease-modifying agents. The DMD market will grow as a result of polytherapy as patients … Continue reading →

CHATTANOOGA, Tenn.--(BUSINESS WIRE)-- Four dads whose sons have Duchenne muscular dystrophy will climb Clingmans Dome, the highest peak in the Smoky Mountains, on June 30 as part of the annual Climb to CureDuchenne: Pick Your Peak event happening nationwide. People across the country are climbing a mountain, hill or tall building to increase awareness and raise funds to find a cure for Duchenne muscular dystrophy. CureDuchenne, a nonprofit organization that raises awareness and funds research to cure Duchenne muscular dystrophy, organized the event. Todd Crawford, Nick Ware, Jimmy Dotson, and Brad Nance, are Duchenne dads climbing for their sons Mackenzie, 12, Alex, 16, Luke, 7 and Caleb, 6. It is statistically remarkable that four dads, who all work in the Chattanooga information technology industry, all have sons living with Duchenne, a rare and devastating muscle disease that affects 1 in 3,500 boys. Crawford and Dotson both work at Unum; Ware works for Lawson and is a contractor at Unum; and Nance works for Northwest Georgia Bank in the IT department. Crawford created a Climb to CureDuchenne team called CALM Dads, which represents the first initial of each of their sons names, to raise much needed funding for scientific research to … Continue reading →

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Catabasis Pharmaceuticals, a clinical stage biopharmaceutical company dedicated to the discovery and development of innovative, effective and safe medicines for the treatment of inflammatory and metabolic diseases, today announced that Michael Jirousek, Ph.D., chief scientific officer and co-founder of Catabasis, will present at the Parent Project Muscular Dystrophy (PPMD) Annual Connect Conference to be held June 28-July 1, 2012 at the Harbor Beach Marriott Hotel in Ft. Lauderdale, Fla. Dr. Jirousek will present in a research panel focused on the role of inflammation and fibrosis in Duchenne Muscular Dystrophy (DMD) at 4:00 p.m. ET on Saturday, June 30, 2012. This interactive panel is part of a special three-part series being presented at this years meeting focused on clinical trials and the future of this research and treatment for DMD. These panels will feature commentary and insight from parents whose children are or were participating in trials, clinicians who run the trials and from the industry leaders developing these trials. Dr. Jirousek has also been invited to be a guest speaker in a webinar hosted by PPMD on June 6, 2012 at 1 p.m. ET to discuss the new approaches to treating inflammation in DMD. The webinar can … Continue reading →

OXFORD, UNITED KINGDOM--(Marketwire -05/25/12)- Summit (SUMM.L), a UK drug discovery company, today announced that it has dosed the first cohort of patients in a Phase 1 study of SMT C1100 for the treatment of Duchenne Muscular Dystrophy (DMD), a fatal, rare genetic disease characterized by rapidly worsening muscle weakness. SMT C1100, an oral small molecule compound, is a potential disease-modifying drug that works to increase, or upregulate, the amount of a naturally occurring protein called utrophin. "There is currently no known cure for DMD, and the only treatments available mask the symptoms of the disease," said Glyn Edwards, Chief Executive Officer of Summit. "SMT C1100 has the potential to modify the underlying disease, and the initiation of this Phase 1 trial represents a great step forward in bringing our breakthrough science to patients suffering from DMD. We expect to report top-line data from the full trial before the end of this year." SMT C1100 has been extensively evaluated in non-clinical efficacy and safety studies and has demonstrated its ability to restore and maintain the function of muscles. This Phase 1 dose-escalating clinical trial in healthy volunteers will evaluate if the Company's aqueous formulation of SMT C1100 can provide the consistent … Continue reading →

Registry bid to tackle devastating condition 8:00am Saturday 19th May 2012 in News By Barry Nelson, Health Editor THOUSANDS of people affected by a form of muscular dystrophy are being invited to sign up to a new national registry based in the North-East which could help lead to a treatment or cure. The initiative is being backed by Blaydon MP Dave Anderson, who has lost his sister, brother, niece and nephew to myotonic dystrophy. He said: To tackle myotonic dystrophy effectively, we need the thousands of families affected to speak up. Until now, no database has been available to researchers of people in the UK affected by myotonic dystrophy, an inherited condition, which causes muscles to waste, weaken and stiffen, often leading to increasingly severe disability, learning difficulties and shortened life expectancy. The new Newcastle-based registry, funded by the Muscular Dystrophy Campaign and the Myotonic Dystrophy Support Group, will allow clinicians and researchers to speed progress towards a treatment by enabling them to contact people faster, recruit for clinical trials and to better understand the condition. The charities are appealing to the estimated 6,000 people in the UK thought to have Type 1 myotonic dystrophy to come forward and add … Continue reading →

Source: ISNA, Tehran Iranian researcher and lecturer Radbod Darabi jointly with his collogues from the University of Minnesota's Lillehei Heart Institute have effectively treated muscular dystrophy in mice using human stem cells derived from a new process which for the first time makes the production of human muscle cells from stem cells efficient and effective. Radbod Darabi, MD, PhD with Rita Perlingeiro, PhD. (Credit: Image courtesy of University of Minnesota Academic Health Center) The research outlines the strategy for the development of a rapidly dividing population of muscle-forming cells derived from induced pluripotent (iPS) cells. IPS cells have all of the potential of embryonic stem (ES) cells, but are derived by reprogramming skin cells. They can be patient-specific, which renders them unlikely to be rejected, and do not involve the destruction of embryos. This is the first time that human stem cells have been shown to be effective in the treatment of muscular dystrophy. According to the researchers, there has been a significant lag in translating studies using mouse stem cells into therapeutically relevant studies involving human stem cells. This lag has dramatically limited the development of cell therapies or clinical trials for human patients. The latest research from the … Continue reading →

Researchers from the University of Minnesota's Lillehei Heart Institute have effectively treated muscular dystrophy in mice using human stem cells derived from a new process that for the first time makes the production of human muscle cells from stem cells efficient and effective. The research, published today in Cell Stem Cell, outlines the strategy for the development of a rapidly dividing population of skeletal myogenic progenitor cells (muscle-forming cells) derived from induced pluripotent (iPS) cells. iPS cells have all of the potential of embryonic stem (ES) cells, but are derived by reprogramming skin cells. They can be patient-specific, which renders them unlikely to be rejected, and do not involve the destruction of embryos. This is the first time that human stem cells have been shown to be effective in the treatment of muscular dystrophy. According to U of M researchers who were also the first to use ES cells from mice to treat muscular dystrophy there has been a significant lag in translating studies using mouse stem cells into therapeutically relevant studies involving human stem cells. This lag has dramatically limited the development of cell therapies or clinical trials for human patients. The latest research from the U of M … Continue reading →

Public release date: 3-May-2012 [ | E-mail | Share ] Contact: Caroline Marin crmarin@umn.edu 612-624-5680 University of Minnesota Academic Health Center MINNEAPOLIS/SAINT PAUL (May 4, 2012) Researchers from the University of Minnesota's Lillehei Heart Institute have effectively treated muscular dystrophy in mice using human stem cells derived from a new process that for the first time makes the production of human muscle cells from stem cells efficient and effective. The research, published today in Cell Stem Cell, outlines the strategy for the development of a rapidly dividing population of skeletal myogenic progenitor cells (muscle-forming cells) derived from induced pluripotent (iPS) cells. iPS cells have all of the potential of embryonic stem (ES) cells, but are derived by reprogramming skin cells. They can be patient-specific, which renders them unlikely to be rejected, and do not involve the destruction of embryos. This is the first time that human stem cells have been shown to be effective in the treatment of muscular dystrophy. According to U of M researchers who were also the first to use ES cells from mice to treat muscular dystrophy there has been a significant lag in translating studies using mouse stem cells into therapeutically relevant studies involving human … Continue reading →

ScienceDaily (May 4, 2012) Researchers from the University of Minnesota's Lillehei Heart Institute have effectively treated muscular dystrophy in mice using human stem cells derived from a new process that -- for the first time -- makes the production of human muscle cells from stem cells efficient and effective. The research, published May 4 in Cell Stem Cell, outlines the strategy for the development of a rapidly dividing population of skeletal myogenic progenitor cells (muscle-forming cells) derived from induced pluripotent (iPS) cells. iPS cells have all of the potential of embryonic stem (ES) cells, but are derived by reprogramming skin cells. They can be patient-specific, which renders them unlikely to be rejected, and do not involve the destruction of embryos. This is the first time that human stem cells have been shown to be effective in the treatment of muscular dystrophy. According to U of M researchers -- who were also the first to use ES cells from mice to treat muscular dystrophy -- there has been a significant lag in translating studies using mouse stem cells into therapeutically relevant studies involving human stem cells. This lag has dramatically limited the development of cell therapies or clinical trials for human … Continue reading →