Category Archives: Muscular Dystrophy Treatment

Newport Beach, Calif., Feb. 21, 2012 (GLOBE NEWSWIRE) -- CureDuchenne, a nonprofit organization that raises awareness and funds multiple research and development projects to cure Duchenne muscular dystrophy, announces the funding of three new research projects following its third annual CureDuchenne Scientific Summit. CureDuchenne is dedicated to finding treatments and a cure for Duchenne regardless of the specific genetic mutation by advancing drug development programs, identifying new drug targets that exhibit potential to transform the treatment of Duchenne and help drive the most promising research programs to market. CureDuchenne's comprehensive research portfolio now funds three new projects that includes development of a new MDX mouse model with duplicated exons, a study with currently approved drugs that may help treat critical cardiac issues and also supports the reformulation of the utrophin upregulator SMT C1100. "CureDuchenne takes a comprehensive and strategic approach to the projects we fund," said Debra Miller, founder and CEO of CureDuchenne. "Every Duchenne child is important so we are funding three promising research projects that address a variety of mutations and could lead to better treatments and hope for Duchenne families." MDX Mouse Model There is currently no animal model with a Duchenne muscular dystrophy duplication and this … Continue reading →

To: HEALTH, MEDICAL AND NATIONAL EDITORS Grant Funds Work of Tivorsan Pharmaceuticals' Team to Bring Recombinant Biglycan to the Clinic for the Possible Treatment of Duchenne Muscular Dystrophy HACKENSACK, N.J., Feb. 21, 2012 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) announced today that it will award Tivorsan Pharmaceuticals (Tivorsan) a $500,000 grant to develop the company's biglycan therapeutic candidate for Duchenne muscular dystrophy. (Logo: http://photos.prnewswire.com/prnh/20100119/DC39975LOGO) Dr. Justin Fallon of Brown University, the founding scientist of Tivorsan, was the recipient of PPMD's first End Duchenne Grant in 2008. Building on that work, the Tivorsan team seeks to use recombinant human biglycan (rh-BGN) to increase utrophin at the muscle cell membrane, resulting in reduced muscle damage and improved function. Utrophin is a molecule that is related to dystrophin in structure and form and can "stand in" for dystrophin when present in greater than normal quantities. Biglycan is a naturally occurring protein made up of amino acids and carbohydrate chains that is found in large amounts on the outside of developing and regenerating muscle cells. The form of biglycan that is active therapeutically contains only simple carbohydrate side chains. For this reason, rh-BGN is straightforward to manufacture. PPMD President and CEO Pat Furlong … Continue reading →

Xiara Leiva, a swim instructor at the California Aquatic Therapy & Wellness Center in Long Beach, works with Joey Perez, l3, who has autism as well as muscular dystrophy and is terminally ill. (Sean Hiller / Staff Photographer) As instructors lift Joey from his wheelchair and place him in the warm pool water, a remarkable transformation takes place. In the wheelchair, Joey looks disinterested, almost like he is bored with his surroundings. But as the warm water envelops his body, his face breaks into a broad, joyful smile. He lets out a cry of pleasure. With the help of lifeguard instructor Xiara Leiva and a flotation device, Joey starts to move legs that had been rigidly locked together in his wheelchair. He lays his head back in his water world of freedom, happy as a 13-year-old boy suffering from severe autism and muscular dystrophy can be. "He comes to life in the pool. He speaks with his eyes," says Pat Dixon, executive director Patricia Dixon, executive director at the California Aquatic Therapy & Wellness Center, enjoys the company of parent Mieko Hester-Perez, while her son Joey Perez, l3, who has autism and muscular dystrophy, enjoys the warm-water pool therapy in … Continue reading →

BY CHELSEA JENSEN WEST HAWAII TODAY cjensen@westhawaiitoday.com Shriners Hospital for Children is headed to the Big Island to connect pediatric doctors with young patients who need their specialized care. The Honolulu-based hospital, which serves Hawaii and the Asia and Pacific regions, will have outreach clinics at two West Hawaii locations Feb. 27 through 29, said Sandy Zukeran, the nonprofit hospital's outreach coordinator. The clinics are held on Hawaii Island semiannually, offering neighbor island residents an opportunity for children under age 18 to see specialized doctors without making the trip to Oahu, said Dr. Craig Ono, the hospital's interim chief of staff. The hospital has three pediatric surgeons on staff. "We are trying to enhance the level of care we provide throughout the state of Hawaii for pediatric work," Ono said, noting just one other pediatric surgeon outside the children's hospital travels to the outer islands. "There are no others. We're the pediatric surgeons who are sub-specialized in taking care of the musculoskeletal problems in children." The first clinic is slated for 8 a.m. to 4 p.m. Feb. 27 at the Hilton Waikoloa Village, said Zukeran, who confirmed the Hilton will validate parking for visiting patients. Clinics will also be held … Continue reading →

The protein biglycan needs to be present to stabilize synapses at the neuromuscular junction after they have formed, according to research led by Brown University that appears in the Feb. 14, 2012, issue of the Journal of Neruoscience. "What neuromuscular junctions do second-by-second is essential for our brain to control movement and they are also important for the long-term health of both muscle and motor neurons," said Justin Fallon, profesor of neuroscience at Brown University and the paper's senior author. "A treatment that sustains or supports the synapse could promote the health of motor neurons and muscle." In previous work, Fallon, a member of the Brown Institute for Brain Science, has shown that in mice with the same genetic mutation as Duchenne patients, biglycan promotes the activity of another natural protein, utrophin, that can significantly reduce the muscle degradation that patients suffer. Utrophin essentially takes over for dystrophin, which is the protein Duchenne patients cannot produce. In 2010 Brown licensed Fallon's biglycan intellectual property to the Providence startup company Tivorsan Pharmaceuticals, which is working toward human trials of biglycan. Last month the Muscular Dystrophy Association, which helped support Fallon's new study, gave Tivorsan a $1 million grant. Now Fallon's research … Continue reading →

CLEVELAND, Feb. 14, 2012 /PRNewswire/ -- Milo Biotechnology will receive $250,000 from JumpStart Inc., a nonprofit investing intensive business assistance and some capital into early stage Northeast Ohio-based tech companies.  A clinical stage startup developing a therapy to increase muscle strength and improve the quality of life of muscular dystrophy patients, Milo is the 61st company in JumpStart's portfolio.  "There are limited treatment options for muscular dystrophy and approved therapies are inadequate or can cause significant side effects," says JumpStart's Mike Lang. "Since most muscular dystrophy patients are children, the medical community is interested in a safer and more targeted therapy." Milo Biotechnology's lead product is an adeno-associated virus (AAV) delivered follistatin protein.  Follistatin inhibits the activity of myostatin, a protein that impedes muscle differentiation and growth.  That makes follistatin a very potent stimulator of muscle growth and prevention of muscle scarring after injury.  "The translational research team at the Research Institute at Nationwide Children's Hospital has harnessed the power of follistatin and increased its specificity for muscle," explains Al Hawkins, CEO of Milo Biotechnology and CEO-in-Residence at BioEnterprise in Cleveland. "The efficacy and safety results thus far have been remarkable." Pre-clinical studies in mice and non-human primates demonstrated considerable increases … Continue reading →

Newswise — Linda H. Cripe, MD, a distinguished pediatric cardiologist, has joined the Heart Center at Nationwide Children’s Hospital. Dr. Cripe will also be a member of the faculty at The Ohio State University College of Medicine. Dr. Cripe is well-known for her continuing work in studying the care and treatment of cardiomyopathy associated with neuromuscular disease, especially Duchenne muscular dystrophy (DMD). She was a member of the Centers for Disease Control (CDC) National Steering Committee – Duchenne Muscular Dystrophy Standards of Care, and has been an invited lecturer nationally and internationally on cardiomyopathy related to DMD. She currently is a member of the Scientific Advisory Board for Parent Project Muscular Dystrophy. Dr. Cripe was most recently the Chair of the Reappointment, Promotion and Tenure Committee and Coordinator for Medical Student Education in the Division of Cardiology at Cincinnati Children’s Hospital Medical Center and served as an Associate Professor of Pediatrics at the University of Cincinnati. She completed her residency at the University of Iowa Hospitals and Clinics, and served as a pediatric cardiology fellow at the University of Iowa Hospitals and Clinics and at Children's Hospital Boston. She also has held faculty appointments at The Children's Hospital in Denver, … Continue reading →

20-01-2012 04:32 http://www.telethon.it - Pier Lorenzo Puri is a researcher at the Telethon Dulbecco Institute and works at the Santa Lucia Foundation in Rome. After a number of years as a researcher in the United States, he returned to Italy thanks to Telethon offering him a chance to create his own independent research laboratory in Italy in 2001.Having graduated as a medical doctor, Pier Lorenzo studies Duchenne muscular dystrophy and in particular the stage of the disease during which the patient's muscles try and mend themselves using particular stem cells. By intervening pharmacologically on this mechanism, Puri and his group aim to prolong for as long as possible this phase in which patients are still able to move autonomously and are above all more receptive to eventual treatment currently being studied. Read more: "For treatment we will have in the future" - Video … Continue reading →

To: HEALTH, MEDICAL AND NATIONAL EDITORS TUCSON, Ariz., Feb. 1, 2012 /PRNewswire-USNewswire/ -- The Muscular Dystrophy Association's latest round of grants allocates more than $12 million to the support of 38 research projects investigating the causes of, and potential treatments for, a number of forms of neuromuscular disease. "MDA is strongly committed to funding important and exciting basic research that will serve as the basis of future therapies for neuromuscular disease," said MDA Chairman of the Board R. Rodney Howell, M.D. Howell is a professor of pediatrics in the Miller School of Medicine at the University of Miami (Fla.). The new grants, which became effective Feb. 1, support research into more than 15 diseases in MDA's program, including Duchenne muscular dystrophy (DMD), ALS (amyotrophic lateral sclerosis or Lou Gehrig's disease), spinal muscular atrophy (SMA), facioscapulohumeral muscular dystrophy (FSHD), and others. MDA Grants Set Sights on Therapies In addition to funding investigations into underlying disease mechanisms, the grants will help guide the development of strategies for diagnosis and treatment. For example, in Pompe disease (acid maltase deficiency), scientists will look for ways to improve the current FDA-approved treatment -- enzyme replacement therapy -- which sometimes leads to an unwanted immune response. … Continue reading →

To: HEALTH, NATIONAL AND RETAILING EDITORS Leading Duchenne Muscular Dystrophy Organization to Receive Donation from The Caring Valley(TM) HACKENSACK, N.J., Feb. 1, 2012 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) announced today that it has been selected as a featured charity in The Caring Valley, an area on the Webkinz World(TM) site that teaches children about child-focused nonprofits in a fun environment. PPMD Founding President and CEO was thrilled that Duchenne is receiving this kind of national exposure from the Webkinz World site. "Webkinz Foundation's dedication to helping leading children's organizations is admirable. We are humbled and appreciative to have been chosen. The awareness that being part of The Caring Valley in Webkinz World will bring Duchenne muscular dystrophy is immeasurable. Our families know Webkinz and love this brand, and now they will be grateful to this brand for their dedication to our patient community." Each of the sections in The Caring Valley contains information about different child-focused nonprofits. Children learn about the organizations as they play and pick where they want to plant their virtual saplings. Webkinz Foundation will donate to each charity. Charities featured in The Caring Valley are selected to reflect the broad range of issues supported … Continue reading →