Category Archives: Muscular Dystrophy Treatment

March 31, 2013 Back Home! By: Judi Lecoq … Continue reading →

By Zeba Siddiqui (Reuters) - After three decades without bringing a drug to market, Sarepta Therapeutics Inc stands on the verge of a breakthrough with its treatment for a crippling genetic disorder that affects one in every 3,500 newborn boys. If U.S. regulators fast-track approval of its treatment for Duchenne muscular dystrophy, as some investors are betting, it would complete a remarkable turnaround for the company that began life as AVI Biopharma in Corvallis, Oregon, 33 years ago. Sarepta's stock has more than doubled in value since October, when its drug, eteplirsen, yielded positive results in a mid-stage trial. The company has a market capitalization of $1.1 billion. Duchenne muscular dystrophy, or DMD, is classed as an orphan disease -- a condition affecting fewer than 200,000 people. More than a quarter of the 39 new medicines approved in the United States last year were designated for such diseases. It's an area that has grabbed the attention of drug developers in search of a unique product that can command a high price. There are no approved treatments for DMD. "Sarepta has the product and development platform necessary to join the ranks of other successful companies that target rare diseases," William Blair … Continue reading →

Public release date: 1-Apr-2013 [ | E-mail | Share ] Contact: Jillian Hurst press_releases@the-jci.org Journal of Clinical Investigation Duchenne muscular dystrophy (DMD) is a degenerative skeletal muscle disease caused by mutations in the protein dystrophin. Dystrophin functions to protect muscle cells from injury and loss of functional dystrophin results in break down and loss of muscle cells. A cellular signal relay system, known as a MAP kinase cascade, regulates the function of muscle stem cells, serving as a source of the new cells that are required for muscle regeneration. In this issue of the Journal of Clinical Investigation, researchers led by Anton Bennett at Yale University identified the protein MKP-5 as a negative regulator of MAP kinase cascades in muscle stem cells. Loss of MKP-5 in a mouse model of DMD enhanced the development of new muscle cells (myogenesis) and helped prevent muscle degeneration. These results identify MKP-5 as an important suppressor of myogenesis and suggest that therapeutics that inhibit MKP-5 could be useful in the treatment of degenerative muscle diseases. ### TITLE: Improved regenerative myogenesis and muscular dystrophy in mice lacking MKP-5 AUTHOR CONTACT: Anton Bennett Yale University School of Medicine, New Haven, CT, USA Phone: 203-737-2441; Fax: 203-737-2738; … Continue reading →

Staying motivated for the Town and Gown run 1:00pm Saturday 30th March 2013 in News By Fran Bardsley, covering Education, East Oxford and Cowley. Call me on 01865 425439 PREPARATIONS are already well underway for this years Oxford Town and Gown run. The May 12 run, backed by the Oxford Mail, will raise money for the Muscular Dystrophy Campaign. Over the next few months we will be following novice runner Kelly Sambrook, 37, who has a personal reason for taking part in the 10k, which makes its way through famous city landmarks. Her nephew, John Dickson, nine, of Bledington, near Chipping Norton, is one of the many youngsters who could benefit from the work done by the charity. Here she talks reporter Fran Bardsley through her second training diary. Im over a month into my training now and starting to feel like Im making real progress. My training app has me running for five minutes between intervals and a total of 15 minutes per session. That may not sound like much but trust me, when youve spent the past 20 years avoiding any form of exercise, its quite an achievement. Im using an interval, or Fartlek (really look it up … Continue reading →

Firefighters will be out on the streets of Little Elm asking motorists to help Fill the Boot for the Muscular Dystrophy Association tomorrow, March 29, next Friday, April 5, and on April 12. Every dollar that passing motorists can pitch into the firefighters boots helps 4, 220 local firefighters provide medical treatment and support services to individuals and their families living with neuromuscular diseases in the Little Elm area. The International Association of Fire Fighters has been a national MDA partner for more than 50 years and remains committed to the fight to end neuromuscular diseases. Fill the Boot funds also are used to support some of the 250 worldwide research projects seeking better treatments and cures for the more than 40 neuromuscular diseases covered by MDA, including Duchenne muscular dystrophy, spinal muscular atrophy and ALS. We know the money we raise definitely makes a difference in the lives of people affected by neuromuscular diseases, and that it helps them get the care they need, said Little Elm Firefighter Clint Long. Fill the Boot is a proud tradition for us, and its a great way for fire fighters to be out in the community reminding residents that we are here, … Continue reading →

NEWPORT BEACH, Calif.--(BUSINESS WIRE)-- CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, has partnered with Dr. Elisabeth Le Rumeur and Pr Jean-Franois Hubert, researchers from the Institute of Genetics and Development of Rennes at the University of Rennes 1 in France, to expand the eDystrophin database. The aim of the partnership is to advance our knowledge about dystrophin mutations found in Becker muscular dystrophy (BMD) patients. eDystrophin is an online database specifically dedicated to providing information about BMD mutations of the DMD gene and the consequences of these alterations from both a clinical and a protein perspective. The database correlates dystrophin structure with clinical outcomes in BMD and will facilitate the understanding of the relationship between the protein and the severity of the disease. Duchenne is a progressive muscle-wasting disease that impacts 1 in 3,500 boys. Boys are usually diagnosed by age 5, in a wheelchair by age 12 and most dont survive their mid-20s. Duchenne, the most common and lethal muscle disease in children, affects 300,000 boys worldwide. Unlike Duchenne, BMD patients display marked differences in their symptoms ranging from asymptomatic to a more severe disease. Only a few studies … Continue reading →

successful treatment for muscular dystrophy treatment in varma massage http://successful-treatment.blogspot.in/p/treatment.html. By: varma4md … Continue reading →

A recent article updated information regarding scoliosis -- curvature of the spine of more than 10 degrees. There are various types of scoliosis. One form is called congenital scoliosis that is due to an abnormality of the vertebrae. As the child grows, the curve can become worse making it more difficult to treat. Neuromuscular scoliosis is associated with certain neurological abnormalities such as muscular dystrophy or spasticity. Scoliosis is also associated with certain diseases such as Marfan syndrome or neurofibromatosis. However, most of the time the cause of the scoliosis is unknown and this is call idiopathic scoliosis. This type of scoliosis is the most common form and is present in about two percent of all adolescents. Fortunately, the majority of times, the curve does not increase to the extent that involved treatment is necessary. Girls are more likely to develop scoliosis than boys. First-degree relatives of individuals with scoliosis are at a greater risk of also developing scoliosis. Adolescents should be examined for curvature of the spine at the childs routine visit to his or her doctor. There are also school-based screening examinations to detect scoliosis. Studies thus far have not shown that such screening examinations result in improved … Continue reading →

New Plush Toy Cures the Smelly Gym Bag, Proceeds Benefit Duchenne Muscular Dystrophy Charity Family invents odor-eliminating toy to Get the ShtankOut, while funding research for deadly disease. ShtankOut owners Marty Karlin and his wife Geri brought the family-invented plush collectibles to market as a way to raise funds for Duchenne research, after their 10-year old son Ryan was diagnosed with the devastating disease. Duchenne is the most common and lethal form of muscular dystrophy, affecting 1 in 3,500 boys worldwide. There is currently no cure or treatment for the disease, in which the muscles wither away, rendering the individual unable to move their legs, arms and hands, eventually attacking the heart and lungs. Boys with Duchenne typically become wheelchair dependent before age 13, with few living beyond their mid-20s. As I watched my sons muscles continue to weaken, it became my lifes work to increase public awareness and help in any way possible to fund research for this tragic disease, says ShtankOut owner Marty Karlin. My family came up with a fun product that solves a smelly problem, and through our donation of proceeds from the sale of ShtankOut for DMD research, we hope to help find a cure … Continue reading →

Mar 27 2013 by Ellie Cullen, Crewe Chronicle THE devoted family of a five-year-old boy are continuing their fundraising mission to help find a cure for his devastating illness. Thomas Robinson, from Crewe, suffers from Duchenne muscular dystrophy, a genetic condition which causes muscles throughout the body to weaken and waste over time. Very few children born with the condition will live to see their 30th birthday. Thomass parents Karen and Neil, and both sets of grandparents, Ken and Margaret Robinson and Kevin and Muriel Henson, have joined forces to raise as much money as possible for the Muscular Dystrophy Campaign. They have organised a concert hosted by the Crewe Male Voice Choir on Saturday, April 6, to help fund research into potential treatments and cures. Thomas was diagnosed with the muscle-wasting condition at just six months old, leaving his entire family heartbroken. Dad Neil said: Weve been told its likely that Thomas will be permanently using a wheelchair in the next five or six years. The way that Duchenne muscular dystrophy will affect every aspect of Thomass life is always in the back of our minds. The Duchenne Breakthrough Research Fund gives us real hope for his future with … Continue reading →