Category Archives: Muscular Dystrophy Treatment

NEWPORT BEACH, Calif.--(BUSINESS WIRE)--RASRx announced today that the United States Food and Drug Administration (FDA) has granted an Orphan Drug Designation (ODD) for its compound RASRx1902 for the treatment of Duchenne Muscular Dystrophy. RASRx1902 is an oral therapy that has shown positive effects on muscle function in animal models of Duchenne. In these models, the RASRx1902 has improved muscle strength and regeneration while decreasing muscle inflammation, degeneration, and necrosis. Through this ODD, RASRx is eligible for financial incentives that can stimulate investment in this program and expedite its regulatory timeline. In concert with CureDuchenne, the company is now transitioning to toxicology studies to advance RASRx1902 towards human clinical trials for the treatment of Duchenne. RASRxs founders worked with the USC Stevens Center for Innovation, the technology transfer office for the University of Southern California, to exclusively license RASRx1902. Funding by CureDuchenne Ventures and a collaborative U.S. Department of Defense research grant with USC has accelerated the preclinical development of this program. Innovation is a core part of USCs culture and we are proud to support Dr. Kathleen Rodgers and RASRx to facilitate the technology transfer of RASRx1902 for DMD, said Michael Arciero, J.D. Director of Technology Commercialization and New Venture, … Continue reading →

Researchers at the National Institutes of Healths National Center for Advancing Translational Sciences (NCATS) and the University of Nevada, Reno School of Medicine (UNR Med) have demonstrated that a drug originally targeted unsuccessfully to treat cancer may have new life as a potential treatment for Duchenne muscular dystrophy (DMD). The candidate drug, SU9516, represents a different kind of approach for treating DMD, a degenerative muscle disease that usually begins in childhood and has no known cure. It is caused by a faulty gene that leads to progressive muscle weakness, with death often occurring around age 25. Rather than trying to fix or replace the broken gene, SU9516 ramps up the muscle repair process, helping reinforce muscle structure. NCATS Chemical Genomics Center Acting Branch Chief Juan Marugan, Ph.D., and UNR Med Professor of Pharmacology Dean Burkin, Ph.D., led a team that screened more than 350,000 compounds to find SU9516, which had been previously developed as a treatment for leukemia. The research demonstrated that this compound improved muscle function in both laboratory and animal DMD models. The results, published recently in Molecular Therapy, may provide a promising approach against the disorder and other muscle-wasting conditions. Those with DMD lack dystrophin, a protein … Continue reading →

June 13, 2017 Dr Craig Harrison and Dr Kelly Walton from the Monash BDI. Credit: Monash University Monash University's Biomedicine Discovery Institute (BDI) researchers have collaboratively developed a therapeutic approach that dramatically promotes the growth of muscle mass, which could potentially prevent muscle wasting in diseases including muscular dystrophy and cancer. The approach, jointly developed with Baker Heart and Diabetes Institute scientists, combines - for the first time - molecules that inhibit three proteins which in turn repress muscle growth. Published this week in the journal Proceedings of the National Academy of Sciences, the scientists found that inhibiting activin A, activin B and myostatin resulted in skeletal muscle mass increase by as much as 150 per cent in preclinical models. Myostatin has long been recognised as the body's major negative regulator of skeletal muscle mass, helping to maintain muscle homeostasis in the body, but creating molecules to target all three related proteins together was a novel approach. "As a result of the study we can now more precisely regulate - and increase - muscle mass in the setting of disease," co-lead author from Monash BDI, Dr Craig Harrison, said. Dr Harrison said the study, the culmination of many years of … Continue reading →

National Institutes of Health (press release) Researchers aim to repurpose former experimental cancer therapy to treat muscular dystrophy National Institutes of Health (press release) ... Sciences (NCATS) and the University of Nevada, Reno School of Medicine (UNR Med) have demonstrated that a drug originally targeted unsuccessfully to treat cancer may have new life as a potential treatment for Duchenne muscular dystrophy (DMD). and more » Originally posted here: Researchers aim to repurpose former experimental cancer therapy to treat muscular dystrophy - National Institutes of Health (press release) … Continue reading →

Steve Tarter Journal Star city of Peoria reporter @SteveTarter PEORIA At age 33, Travis Platt calculates he has outlived his life expectancy three times. He is making the most of every year he has. Platt, 33, is the owner of Platts Printing in Farmington. His company, which earlier this year opened a second office in the Peoria NEXT Innovation Center, 801 W. Main St., is starting to make a creative splash in central Illinois with advertising and promotional concepts for well-known area entities such as Hy-Vee supermarket, the Peoria Chiefs and Wildlife Prairie Park. But Platt isn't just dealing with a competitive business world. Diagnosed with muscular dystrophy when he was 18 months old, Platt has used a wheelchair since the first grade. "When I wake up in the morning, I have more challenges than most," said Platt, who uses his disability as a motivation to do what he likes to do at high speed. "I don't have unlimited time. I realize I have to do it now," he said. "I was initially told that I wouldn't live past the age of 8. Then, that I might make it to 16. I figure I've outlived my life expectancy three times." … Continue reading →

About Duchenne Muscular Dystrophy Primarily affecting males, Duchenne muscular dystrophy (DMD) is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-twenties due to heart and respiratory failure. It is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of skeletal, diaphragm, and heart muscles. Patients with DMD can lose the ability to walk as early as age ten, followed by loss of the use of their arms. DMD patients subsequently experience life-threatening lung complications, requiring the need for ventilation support, and heart complications in their late teens and twenties. It is estimated that a nonsense mutation is the cause of DMD in approximately 13 percent of patients. About ataluren (Translarna)Ataluren, discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is … Continue reading →

Comedian and host of HBOs Real Time Bill Maher made an impassioned plea for an end to experiments on dogs at Texas Agricultural and Mechanical University (TAMU) that he views as cruel and inhumane. These dogs, he said, are afflicted with canine muscular dystrophy. Thats right, Maher said, Dr. Joe Kornegay is making dogs sick with a debilitating and incurable disease that ravages their bones and bodies on purpose. The dogs struggle to walk as their muscles weaken and waste away. Puppies that would normally be running and playing and digging up rose bushes become completely crippled before they reach six months old. Kornegays research is aimed at finding treatment and possible cures for Duchenne muscular dystrophy. According to his groups website, the National Center for Canine Models of Duchenne Muscular Dystrophy (NCDMD), For the past 25 years, Dr. Kornegay has studied a spontaneous canine disease termed golden retriever muscular dystrophy (GRMD), which serves as an animal model for Duchenne muscular dystrophy (DMD) of humans. Both conditions are X-linked, occurring due to mutations in the dystrophin gene. Through his work with canines, Kornegay is attempting to improve life for the 1 out of every 3,500 males born with Duchenne Muscular … Continue reading →

The FDA has handed out a Fast Track Designation to AMO Pharma for its key asset AMO-02 to treat congenital myotonic dystrophy. The British-American biotech hybrid AMO Pharma is advancing asmall pipeline of drugs for rare genetic disorders. The biotech, which is backed by famous UK Investor Neil Woodford, is currently pushing its lead candidate AMO-02 through Phase II as the first-ever treatment for myotonic dystrophy. Now, AMO will also be backed by regulatory agencies, after the FDA today announced that it has granted Fast Track Designation for AMO-02. The Fast Track Designation paves the way for an accelerated approval process for AMOs lead drug and includes a priority review that clips four months off the FDAs decision process. This is great news for patients suffering from the disease, which represents the most common form of muscular dystrophy, and for which there is currently no approved therapy available. AMO-02 tries to tackle the most severe form of myogenic dystrophy known as congenital DM1. The drug works by inhibiting theglycogen synthase kinase 3 beta (GSK3), which is extra active in patients with DM1 as well as duchenne muscular dystrophy. So far, theinhibitor was able to demonstrate pre-clinical efficacy in transgenic models. … Continue reading →

NEWPORT BEACH, Calif.--(BUSINESS WIRE)--CD Access is a new nonprofit that provides patients access to drugs and therapies that are currently unavailable through clinical trial or prescription. CD Access first program allows Duchenne muscular dystrophy patients in Canada that previously participated in a clinical trial for drisapersen to begin redosing through a Health Canada Special Access Programme. BioMarin transferred their supply of exon 51 skipping drug, drisapersen, to CD Access for distribution to boys who were participants in previous clinical trials in Canada. Former trial participants in Canada will need to contact their physician to confirm eligibility. The drug will be administered at Childrens Hospital of Western Ontario, Childrens Hospital of Eastern Ontario, Childrens & Womens Health Centre of BC and potentially one additional site in Canada. We are delighted to provide access to eligible patients who benefited from drisapersen, said Debra Miller, CEO and founder of CureDuchenne and the newly formed CD Access. We believe it is important for Duchenne patients who participated in the clinical trial to have access to investigational drugs when and where there are no approved options for treatments for this rare disease. When BioMarin discontinued development of drisapersen we received multiple calls from parents who … Continue reading →

"Congenital myotonic dystrophy is a devastating neuromuscular disease where affected patients currently have no treatment options available," said Michael Snape, chief executive officer of AMO Pharma. "The designation of Fast Track status for our development program for AMO-02 highlights both the urgent need for a treatment for patients and the potential for this novel therapy to offer a major advance in their care in the years ahead." AMO-02 (tideglusib) is in clinical stage development for the treatment of the severe form of myotonic dystrophy known as congenital DM1. In cellular and animal models of congenital DM1 and Duchenne muscular dystrophy, as well as in muscle biopsies from patients, activity of glycogen synthase kinase 3 beta (GSK3) has been shown to be increased. Inhibitors of GSK3 have been shown to correct the activity of RNA binding proteins, such as CUGBP1, in animal models of DM1. AMO-02 is an inhibitor of GSK3 that has demonstrated pre-clinical efficacy in transgenic models and reversal of muscle cell differentiation deficits in ex vivo tissue samples derived from patients with congenital DM1. "Our ongoing phase 2 clinical trial for AMO-02 in the UK is the first sponsor-led clinical study in the treatment of congenital myotonic dystrophy … Continue reading →