Category Archives: Muscular Dystrophy Treatment

Limb Girdle Muscular Dystrophy | Stem Cell Treatment Patient testimonial of Limb Girdle Muscular Dystrophy treated by StemRx Bioscience Solutions Pvt. Ltd. By: StemRx BioScience … Continue reading →

Caring for Connor is Thanet Extras Charity of the Year for 2015. Connor Olsen is five years old and suffers from Duchenne muscular dystrophy (DMD). Wayne and Georgina Olsen with Kye, two, and Connor, five The condition causes muscle degeneration, which means he could be in a wheelchair by the age of 10, and has a life expectancy of just 20. There is no known cure for the condition and research into treatments is not funded by the NHS. His family want to make sure there is funding to ensure Connor, who goes to St Crispins Infant School, has everything he needs to fight the progressive disease. Connors mum Georgina said: Its fantastic weve been chosen. The support in Thanet and across Kent has always been overwhelming. Connor is ok at the moment, but he is slowing down a little bit now, he struggles up the stairs more and has less energy. He is in Year 1 at school and is really enjoying it. But time is ticking, Connors muscles are wasting, and time goes by so quickly so were hoping people can donate a bit of their time or money to help. A lot of people seem to think … Continue reading →

A Northbrook company said it has won fast-track status for approval of a drug that could become the first in the United States to treat Duchenne muscular dystrophy, a fatal muscle disease found most often in boys. Privately held Marathon Pharmaceuticals said it hopes to win federal approval for deflazacort by 2016. About 20,000 boys and young men in the U.S. live with Duchenne, a progressive disease that's passed by mothers to sons but rarely to daughters, though the daughters can become carriers. It is characterized by a progressive loss of muscle strength, with many losing the ability to walk in their teens. Patients often die from respiratory or heart failure. Deflazacort, a steroid, has been shown to prolong lives as well as extend the ability to walk and breathe. But it is not a cure. It has been available in other countries such as Canada, Mexico and the United Kingdom for decades. U.S. Food and Drug Administration approval is eagerly awaited by people such as Ellen Wagner, whose son, Tim, was diagnosed at 2 with Duchenne. The McHenry mom began ordering deflazacort from the United Kingdom after the physician treating her son wrote a prescription. Its use in the … Continue reading →

A Coventry mum travelled to Downing Street in her bid to get the pioneering drug that could help save her sons life. Jaspal Mann has watched on as her five-year-old son Kirath has battled against muscle-wasting condition Duchenne muscular dystrophy, which leaves many children in a wheelchair before their 10th birthday. Problems with heart and lung muscles also mean that few with the condition currently live to see their 30th birthday. The desperate mum, from Westwood Heath, joined other families from across the country and members of the Muscular Dystrophy Campaign as they handed over a petition to MPs and peers in Westminster calling on the NHS to end delays to a breakthrough treatment called Translarna. The drug is the first ever to tackle the causes of Duchenne muscular dystrophy. It was approved in Europe in August and is currently available in France, Spain, Germany, Italy and Denmark. However, NHS administrative issues have led to agonising delays for families in the UK. Jaspal, 38, said: Kirath loves nothing more than talking to his friends about his favourite super heroes, dinosaurs and playing with his toy cars. Simply put, Kirath just wants to be one of the boys. Although hes still … Continue reading →

Researchers have successfully improved the ability of muscle to repair itself - by artificially increasing levels of the BMI1 gene in the muscle-specific stem cells of mice with muscular dystrophy. The BMI1 gene has been previously linked to the body's ability to regenerate tissue cells in areas such as blood or skin. Led by Queen Mary University of London and published in the Journal of Experimental Medicine, the study provides the first proof of concept that manipulating the activity of this gene enhances the regeneration of the dystrophic muscle to a level where strength is visibly improved. For example, the mice were able to run on a treadmill for a longer time period and at a faster pace. This line of research will now be further developed and scientists aim to one day apply the treatment to patients with chronic muscle wasting such as muscular dystrophy. Muscular dystrophy is a devastating and incurable condition. Duchenne Muscular Dystrophy - the deadliest form of the muscle-wasting disease - is caused by mutations in a gene which eventually cause muscle fibres to become damaged and waste away. Duchenne Muscular Dystrophy is characterised by repeated cycles of muscle damage and repair, resulting in exhaustion … Continue reading →

A FAMILY from Poole calling for the NHS to provide a life-changing drug for their five-year-old little boy are taking their campaign to Downing Street today. Luca Fernades has the muscle-wasting condition Duchenne muscular dystrophy. Many boys with the condition need to use a wheelchair by the time they are eight or nine-years-old and problems with the heart and lung muscles mean few sufferers live to see their 30th birthday. Armed with a petition signed by tens of thousands of people, Lucas parents Pedro and Joanne are heading to Downing Street calling for the government to make a new drug, Translarna, available to Luca and thousands like him. The cutting-edge treatment is already available for boys in other European countries but the family says NHS administrative issues have led to agonising delays for people in the UK. Mrs Fernades said: At the moment Luca has no idea what his life has in store for him. Now he runs, jumps, swims and cycles on his bike, oblivious to it all. However, Lucas condition is starting to affect him more and more each day and we are having to watch our beautiful boy slowly lose his strength. To be eligible for the … Continue reading →

A HAMPSHIRE family will be pounding the streets of London today in a bid to urge health chiefs to fund a life-prolonging treatment for their little boy. Time is running out for seven-year-old Jagger Curtis who suffers from Duchenne muscular dystrophy which means he is unlikely to live beyond his 20s. However, his family were given hope that a new drug on the market could help prolong his life and improve the quality of it. Jaggers mum Jules Geary was hoping that her son would by now be on the drug Translarna after Jagger was chosen as one of only four boys in Southampton who suffers from the particular type of muscle wasting disease that the drug could help. But last month she discovered that funding for the drug had not been approved by NHS England - the body which approves and pays for drugs to be prescribed on the National Health Service. Jules, 39, from Romsey said: We couldnt believe it. For the drug to work Jagger still needs to be mobile, which he is now but that is likely to change by the time he reaches eight or nine. We simply dont have time to wait, this is … Continue reading →

HACKENSACK, N.J., Jan. 12, 2015 /PRNewswire-USNewswire/ --Parent Project Muscular Dystrophy (PPMD), a nonprofit organization dedicated to the fight to end Duchenne muscular dystrophy (Duchenne), announced that John Porter, PhD, former program director of the National Institutes of Health (NIH)/National Institute of Neurological Disorders and Stroke (NINDS) will take on the role of Chief Executive Officer for the organization. For 20 years, PPMD has led the fight to end Duchenne the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 3,500 to 5,000 live male births. The loss of a key muscle protein called dystrophin causes muscle wasting and weakness, eventually leading to the loss of ambulation, difficulty breathing, and heart failure. Death typically occurs in the mid-to late 20s. As program director at the NIH/NINDS, Dr. Porter was responsible for managing a portfolio of research grants across neuromuscular disorders and served as Executive Secretary of the interagency Muscular Dystrophy Coordinating Committee. Prior to that, he served as Professor of Neurology at Case Western Reserve University, where he was supported by the NIH and advocacy groups for research on muscle biology in health and disease, including muscle group responses to Duchenne. "PPMD is beyond thrilled to have … Continue reading →

NEW YORK (TheStreet) -- Shares ofSarepta Therapeutics (SRPT) plunged more than 18% to a 52-week low of $11.33 on Monday after the biopharmaceutical company provided a 168-week update on its study of eteplirsen, the company's Duchenne muscular dystrophy (DMD) treatment. Sarepta's update revealed thatanaccelerated decline in walking ability in the 168th week compared to the previous update after week 144. All the DMD patients lost some walking ability in the trial. However, the update also revealed some encouraging news, as patients in the trialcontinue to walk more than three years after starting eteplirsen treatment. The patients showed no significant side effects or tolerability problems. Eteplirsen also seemed to slow the progression of DMD in the patients. Must Read:Sarepta Updates Eteplirsen Study Results, FDA Filing Still on for Mid Year Jim Cramer and Stephanie Link reveal their investment tactics while giving advanced notice before every trade. Access the tool that DOMINATES the Russell 2000 and the S&P 500. Jim Cramer's protg, David Peltier, uncovers low dollar stocks with extraordinary upside potential that are flying under Wall Street's radar. Read the original here: Sarepta Therapeutics (SRPT) Stock Hits One-Year Low Today After Eteplirsen Update … Continue reading →

Hilo native Daryl Sammy Sampaga is at it again. A year after making headlines in early 2014 for trekking the Big Island-round to raise awareness of muscular dystrophy and to support keiki with disabilities, Sampaga will take on the island of Kauai by two means: walking and paddling. Sampaga will begin the journey, nicknamed Operation Paddle and Walk for Dreams Kauai Island 2015, on Jan. 17. Before I started (the Big Island) walk, I had mentioned to everyone that I was going to hit this island and then hit all the other islands, Sampaga said. They doubted me the first time, saying you not going make em, and no one every tried and I said I will try, even if I got to crawl, I will crawl and I did. The Kauai project, the latest of his nonprofit, Operation Sammy Muscular Dystrophy, will take Sampaga, who has muscular dystrophy, which affects his leg muscles, on a more than 100-mile trip around the Garden Island. The journey will comprise 78 miles of walking and up to 32 miles of paddling along the Na Pali Coast, Sampaga told West Hawaii Today on Tuesday. He will begin the feat first by paddling the … Continue reading →