Category Archives: Muscular Dystrophy Treatment

DUBLIN--(BUSINESS WIRE)--The "Europe Limb-Girdle Muscular Dystrophy Market and Competitive Landscape - 2017" report has been added to Research and Markets' offering. The latest research, Europe Limb-Girdle Muscular Dystrophy Market and Competitive Landscape - 2017, provides comprehensive insights into Limb-Girdle Muscular Dystrophy pipeline, epidemiology, market valuations, product sales, market forecast, product forecasts, and market shares. This study accurately estimates and forecast Limb-Girdle Muscular Dystrophy market size and drug sales. This research also provides insights into Limb-Girdle Muscular Dystrophy epidemiology and late stage pipeline. The report is classified into nine sections - Limb-Girdle Muscular Dystrophy overview with definitions, symptoms, etiology, diagnosis, treatment options; Limb-Girdle Muscular Dystrophy pipeline insights covering late stage clinical trials pipeline; Limb-Girdle Muscular Dystrophy prevalence trends by countries; Limb-Girdle Muscular Dystrophy market size and forecast by countries, market events, trends; product sales and forecast by countries; market shares by countries. The research scope includes EU5 countries - Germany, France, Italy, Spain, UK, Europe. Key Topics Covered: 1. Limb-Girdle Muscular Dystrophy: Disease Overview 2. Limb-Girdle Muscular Dystrophy Pipeline Insights 3. Limb-Girdle Muscular Dystrophy Epidemiology Analysis 4. Germany Limb-Girdle Muscular Dystrophy Market Insights 5. France Limb-Girdle Muscular Dystrophy Market Insights 6. Italy Limb-Girdle Muscular Dystrophy Market Insights 7. Spain Limb-Girdle Muscular … Continue reading →

The sequencing of the canine genome along with next generation sequencing technologies like whole exome sequencing have facilitated quicker, easier and more efficient identification of genes and mutations that can cause diseases in dogs. In a study published in Skeletal Muscle researchers have used these technologies to study a form of Limb-girdle muscular dystrophy (muscle wasting and weakness in shoulder and hip muscles) in Boston terriers. Here to tell us about the research and what this means for the breed is lead author of the study Melissa L. Cox. Melissa L. Cox 5 Sep 2017 Dogs live with humans, and have access to medical care nearly as sophisticated as ours. We are also close in other ways: sharing approximately 85% of our genome that is our complete sets of genes any naturally occurring gene mutation that may cause a disease in dogs is likely to cause a similar condition in humans, and vice versa. Dogs can serve as models of human disease; for example, treatments such as gene therapy can be tried in dogs before going into clinical trials in humans, which can benefit both species. The sequencing of the canine genome greatly increased the speed and efficiency with which … Continue reading →

When Charley Seckler was 3 years old, everyone seemed to notice his big, strong calves. People always said, Charley, youre gonna be a soccer player, his mom, Tracy Seckler, tells PEOPLE. But when Charleys pediatrician said he might have muscular dystrophy, Tracy began to connect the dots. The concerned mother went online and saw videos of other kids who had her sons same big calves, and they were all using their hands to push themselves off the floor just like her son did. She knew instantly that Charley hadDuchenne muscular dystrophy (DMD). Duchenne is a rare, progressive, degenerative disease affecting about 1 in 3,500 boys, according to the Muscular Dystrophy Association (MDA). Children with the condition are born without the protein, dystrophin, which results in muscle degeneration. Eventually, they cant walk, feed themselves or breathe on their own. One of the symptoms is enlarged calf muscles because the muscle tissue is abnormal and may contain scar tissue, according to the MDA. Until recently, boys with DMD usually didnt survive beyond their teen years. Life expectancy is increasing and survival into the early 30s is becoming more common. Tracy and Charley SecklerCourtesy Tracy SecklerAt the time Charley was diagnosed, Tracy who … Continue reading →

GENEVA In what has become a Labor Day weekend tradition, Geneva firefighters will again take part in the annual Fill the Boot campaign to support the Muscular Dystrophy Association (MDA). The Geneva firefighters, representing Local #2859 of the International Association of Firefighters, will collect money in boots to help children and adults with muscular dystrophy, amyotrophic lateral sclerosis (ALS, also known as Lou Gehrigs disease) and related muscle-debilitating diseases live longer and grow stronger. MDA officials said this years Labor Day weekend and Fill the Boot campaign has special meaning, as MDA is mourning the recent passing of longtime national chairman Jerry Lewis, a stalwart of Labor Day weekend MDA telethons. Continuing a more than 60-year tradition, Geneva firefighters will hit the streets with boots in hand, asking motorists, pedestrians and other passersby to donate to MDA. They will be on the corner of North and Exchange streets starting at 10 a.m. Friday. We are thrilled to be working with the Geneva Firefighters IAFF Local #2859 for a Fill the Boot this year to help provide the funds needed to find treatments and cures for muscular dystrophy, ALS and related diseases that severely limit strength and mobility, said Maggie Thomas, … Continue reading →

The chemical structure of a drug can make the entire difference between a success and a failure in drug development. Of two drugs that used the same approach to treat Duchenne muscular dystrophy Exondys 51 (eteplirsen) and Kyndrisa (drisapersen) only one became approved. Researchers from The Ohio State Universityin Columbus now explain how and why the fate of the two seemingly similar drugs separated, in a review published in the journal Expert Opinion on Orphan Drugs. The review was titled Clinical trials of exon skipping in Duchenne muscular dystrophy. Sarepta Therapeutics approved Duchene treatment Exondys 51 is commonly referred to as exon skipping 51 therapy. It aims to correct the underlying genetic flaw in the dystrophin gene, causing Duchenne MD. While researchers know that people can have numerous different mutations that cause the disease, those affecting exon 51 are considered the largest group, present in about 13 percent of patients. A gene in this case that encodes for dystrophin is made up of regions that code for the protein, called exons, as well as noncoding sequences. What has been particularly difficult when developing gene therapies for Duchenne is the extremely large size of the dystrophin gene. With its 79 exons … Continue reading →

Appeal for help: Thomas with wife Catherine Vagappan and James at the hospital. IPOH: The family of a teen suffering from a genetic disorder which causes progressive muscle degeneration and weakness is appealing to the public to help fund his treatment. James Edmund Thomas, 17, who suffers from Duchenne Muscular Dystrophy, has been bedridden in a hospital here for a month after suffering from pneumonia and pulmonary oedema (accumulation of fluid in the lungs) on July 23. James was in intensive care and required a tracheostomy (an opening through the neck) to help him breathe. The medical cost for his treatment was RM130,000. His father Thomas Arokiasamy, 50, has only paid RM80,000 from his savings and with the help of friends. The technician, who is the familys sole breadwinner, said he had no choice but to seek funds from well-wishers. Thomas said his son, who was in a coma at one point, was slowly recovering but could only be discharged if there was a ventilator and other medical equipment at home. He also needs someone to monitor and manage his daily needs. This long-term treatment for James would cost the family between RM65,000 to RM70,000. Although James had been suffering … Continue reading →

Endurance, strength, enthusiasm, persistence those are some of the key themes of the retro-styled Women of the Lynchburg Fire Department calendar that will bring some Lynchburg firefighting flair to peoples walls in 2018 and 2019. Abbey Johnston, a captain whos been with the Lynchburg Fire Department for 13 years, said the 18-month calendar hits a number of highlights for the department. For one, she said its the first time the department has had enough women to feature in a calendar all 14 who were employed at the time of the photo shoots are included in the calendar. Two more have joined the department since. Not everyone was all in for the calendar at first, though. Johnston said some firefighters or their families had reservations about the proposed pin-up nature of the photos. But, as Johnston assured them, the pictures would be fashionable and feminine, yet classy, with a retro, 1950s feel. We wanted it to be respectful, she said. We wanted it to focus on strength, the different strengths that it takes to do the job, and to be honorable to all of our members: retired and current, male and female. Other women featured in the calendar include administrative personnel … Continue reading →

Not everyone found Jerry Lewis funny. Some even found him offensive. But no one can deny the impact he had oncomedy and film over a career that stretched from his early childhood almost until his death Sunday at 91. For 45 years,Lewis played another role many people remember at least as well ashis long list oflight comedies and occasional dramatic parts. Lewis' annual Muscular Dystrophy Associationtelethons weremarathon broadcasts that usually began on Labor Day weekendand ranas long as 211/2 hours. And, like his comedy, the shows were not to everyone's tastes. Some contended that his broadcast and similar fundraisers exploited young disease sufferers. But Lewis' affection for the young people he called Jerry's kids was genuine, and many, many people were moved to donate: Over the years, Lewis raised more than $2billion for research into treatment for muscular dystrophy and related diseases. We are deeply saddened by the death of humanitarian and MDA Telethon star and national chairman, Jerry Lewis, Northern Indiana MDA Executive Director Sonja Cronin said in an email Monday. MDA would not be the organization it is today if it were not for Jerry's tireless efforts on behalf of 'his kids.' His enthusiasm for finding cures for … Continue reading →

Written by PETA | August 21, 2017 The late Jerry Lewis Muscular Dystrophy Association telethon may have been one of televisions longest-running and best-known fundraisers, but not everyone was a fan of the program. According to The Washington Post, many muscular dystrophy (MD) patients, as well as some former telethon poster children, cheered when it went off the air in 2010, because reportedly Lewis treated the childrenhe claimed to be helping with little respect, he pitied those living with muscular dystrophy and he used offensive language when describing them. Today, many people with MD are similarly outraged over the cruel MD experiments on dogs being carried out in their name at Texas A&M University (TAMU). After PETA revealed that experimenters at the school intentionally breed dogs to suffer from a severe form of canine muscular dystrophycausing their muscles to weaken and waste away until they can barely walk, swallow, or even breatheMD patients spoke out. I can tell you that I wouldnt wish [MD] on my worst enemy, let alone on defenceless animals, said one MD sufferer named Ian Hughes. These cruel experiments arent workingtheres still no cure or effective treatment for MD. But dont misunderstand me: The choice is … Continue reading →

GODFREY Firefighters of the Godfrey Fire Protection District and Shivers Frozen Custard are partnering to fight muscular dystrophy through a fill-the-boot event on Saturday, Aug. 26. Muscular dystrophy, amyotrophic lateral sclerosis, and other related life-threatening diseases take away peoples ability to walk, move, smile, talk and even breathe. Godfrey firefighter and MDA coordinator Chris Dennison reached out to Shivers Frozen Custard owner Brian Morris about the possibility of a joint event to raise awareness and funds for the Muscular Dystrophy Association. Mr. Morris was very supportive of the idea and we look forward to working with Shivers to help fight muscular dystrophy, Dennison said. The fire district is encouraging residents to stop by Shivers, 3318 Godfrey Road, from 6-9 p.m. Saturday, Aug. 26. Firefighters will be collecting for MDA, all children will receive a free toy fire truck, and a real GFPD firetruck will be on display. Funds raised through the 2017 Godfrey Fire Fill the Boot event will be used to build on decades of research progress, helping push science to its limits in the search for treatments and cures. Over the years, Godfrey firefighters have raised tens of thousands of dollars for the Muscular Dystrophy Association with the … Continue reading →