Category Archives: Muscular Dystrophy Treatment

Diagnosis Your doctor is likely to start with a medical history and physical examination. After that, your doctor may recommend: There's no cure for any form of muscular dystrophy. But treatment can help prevent or reduce problems in the joints and spine to allow people with muscular dystrophy to remain mobile as long as possible. Treatment options include medications, physical therapy, and surgical and other procedures. Your doctor may recommend: Several types of therapy and assistive devices can improve quality and sometimes length of life in people who have muscular dystrophy. Examples include: Surgery may be needed to correct a spinal curvature that could eventually make breathing more difficult. Explore Mayo Clinic studies testing new treatments, interventions and tests as a means to prevent, detect, treat or manage this disease. Respiratory infections may become a problem in later stages of muscular dystrophy. It's important to be vaccinated for pneumonia and to keep up to date with influenza shots. Dietary changes haven't been shown to slow the progression of muscular dystrophy. But proper nutrition is essential because limited mobility can contribute to obesity, dehydration and constipation. A high-fiber, high-protein, low-calorie diet may help. A diagnosis of muscular dystrophy can be extremely … Continue reading →

Definition The muscular dystrophies (MD) are a group of more than 30 genetic diseases characterized by progressive weakness and degeneration of the skeletal muscles that control movement. Some forms of MD are seen in infancy or childhood, while others may not appear until middle age or later. The disorders differ in terms of the distribution and extent of muscle weakness (some forms of MD also affect cardiac muscle), age of onset, rate of progression, and pattern of inheritance.DuchenneMD is the most common form of MD and primarily affects boys. It is caused by the absence of dystrophin, a protein involved in maintaining the integrity of muscle. Onset is between 3 and 5 years and the disorder progresses rapidly. Most boys are unable to walk by age 12, and later need a respirator to breathe. Girls in these families have a 50 percent chance of inheriting and passing the defective gene to their children. Boys withBeckerMD (very similar to but less severe than Duchenne MD) have faulty or not enough dystrophin.FacioscapulohumeralMD usually begins in the teenage years. It causes progressive weakness in muscles of the face, arms, legs, and around the shoulders and chest. It progresses slowly and can vary in … Continue reading →

The Muscular Dystrophy Association (MDA) is an American organization which combats muscular dystrophy and diseases of the nervous system and muscular system in general by funding research, providing medical and community services, and educating health professionals and the general public. Comedian, actor, filmmaker and singer Jerry Lewis was the most popular and well known celebrity associated with MDA, whose work with the organization was known through his annual telethon that he hosted annually from 1966 to 2010 as well as his tenure as its national chairman until 2011. Other celebrities and entertainers have supported the organization over the years, including Dean Martin, Wayne Newton, Frank Sinatra, Michael Jackson, Sammy Davis, Jr., Don Rickles, Ed McMahon, Milton Berle, Norm Crosby, Don Francisco, Aretha Franklin, Maureen McGovern and Diana Ross.[2] MDA's national office is in Chicago, Illinois. The organization was founded in 1950 by a group with personal connections to muscular dystrophy. Originally known as the Muscular Dystrophy Associations of America, it was renamed to its present name in the 1970s.[2] Each year (sometimes for multiple-year stretches), a child affected by a muscle disease is chosen to be the MDA's "National Goodwill Ambassador", which, until the 1980s, were referred to as "poster … Continue reading →

Check out which companies are making headlines before the bell: Exxon Mobil UBS upgraded the stock to "neutral" from "sell," noting the underperformance of the stock this year and an improved macro outlook for the oil industry. Sarepta Therapeutics The drugmaker announced positive study results for a new treatment for Duchenne Muscular Dystrophy. Navistar The truck and engine maker earned 37 cents per share for its latest quarter, beating estimates by nine cents a share. Revenue also topped forecasts. The return to profitability for Navistar comes as it increases market share and profit margins. HD Supply Holdings The industrial distributor matched forecasts with adjusted quarterly profit of 64 cents per share, with revenue very slightly above Street projections. Newell Brands The consumer products maker cut its full-year forecast, due to the impact of Hurricane Harvey on its resin suppliers. Newell's Rubbermaid division is among its substantial users of resins. Trivago The travel website operator cut its full-year outlook, based on slower-than-expected revenue growth. Trivago said revenue per qualified referral a key metric fell too quickly to prevent overspending on ad purchases. G-III Apparel The manufacturer of licensed apparel lost 18 cents per share for its latest quarter, eight cents a … Continue reading →

Shares of Sarepta Therapeutics soared 12 percent in early trading Wednesday after the biopharmaceutical company reported positive results from a clinical trial of an experimental medicine for Duchenne muscular dystrophy. The drug, golodirsen, would be Sarepta's second to treat the rare, genetic disease, which causes muscle wasting and can be fatal before patients turn 30. Sarepta focuses on the discovery and development of precision genetic medicines to treat rare neuromuscular diseases. The new study, conducted in Europe, involved 25 boys with confirmed deletions of the DMD gene amenable to skipping exon 53. Exons are part of the DNA code. The treatment targets a genetic mutation affecting about 8 percent of patients with DMD. Sarepta's first drug for DMD, Exondys 51 approved on a conditional basis by the FDA last year pending more testing to confirm results treats a mutation affecting about 13 percent. Exondys 51 costs about $300,000 per year. "Our goal is to treat 100 percent" of DMD suffers, Sarepta CEO Doug Ingram told CNBC's "Squawk Box." "The data that we have this morning shows we're on the right path." The results, announced before Wall Street's open bell, showed that golodirsen increased production of the protein dystrophin to 1.02 … Continue reading →

Toward the end of trading Wednesday, the Dow traded up 0.42 percent to 21,845.34 while the NASDAQ climbed 0.41 percent to 6,401.95. The S&P also rose, gaining 0.42 percent to 2,468.27. Leading and Lagging Sectors Wednesday afternoon, the energy shares surged 1.23 percent. Meanwhile, top gainers in the sector included Frontline Ltd. (NYSE: FRO), up 5 percent, and JA Solar Holdings Co., Ltd. (ADR) (NASDAQ: JASO), up 6 percent. In trading on Wednesday, utilities shares fell 0.25 percent. Meanwhile, top losers in the sector included NRG Energy Inc (NYSE: NRG), down 3 percent, and Entergy Corporation (NYSE: ETR) down 1 percent. Top Headline G-III Apparel Group, Ltd. (NASDAQ: GIII) reported stronger-than-expected results for its second quarter and raised its FY18 forecast. G-III Apparel reported a Q2 adjusted loss of $0.15 per share on revenue of $538 million. However, analysts were expecting a loss of $0.26 per share on sales of $522 million. Equities Trading UP Verastem Inc (NASDAQ: VSTM) shares shot up 30 percent to $5.00 after the company disclosed that its Phase 3 DUO study evaluating the efficacy and safety of duvelisib showed statistically significant improvement. Shares of Voyager Therapeutics Inc (NASDAQ: VYGR) got a boost, shooting up 20 … Continue reading →

A single gene therapy that silences the mutation responsible for oculopharyngeal muscular dystrophy (OPMD) and replaces the mutated gene with a normal one may advance into human studies in the second half of 2018. Benitec Biopharma started its OPMD program in 2014 and now announced their clinical candidate BB-301 as a one-step gene therapy approach. OPMD patients develop muscle weakness in the upper eyelids and throat in adulthood, typically after age 40. OMPD is a rare genetic disease caused by a mutation of the poly(A)-binding protein nuclear 1 (PABPN1) gene. Because it affects fewer than 200,000 people nationwide, OMPD is considered an orphan disease that benefits from encouraging programs for drugs targeting these rare diseases. In collaboration with research groups in London and Paris, Benitec tested a genetic approach known as DNA-directed RNA interference (ddRNAi) to shut down and replace the mutant PABPN1 gene using two different viral vectors. In this pre-clinical study, researchers found that the two-vector system restored muscular function in A17 mouse model, which displays many OPMD clinical signs including fibrosis and loss of muscle strength. Now, Benitec combined silence and replace gene functions into a single vector (a carrier system) ina new clinical candidate, BB-301. Using … Continue reading →

For more than four months, Bethlehem blogger Shane Burcaw waited anxiously for a call about his future. Whenever my phone rings with the hospitals number, my body goes into complete panic nervous sweats, rapid heart rate as I prepare for what could either be the best or worst news of my entire life. Burcaw wrote on his blog, Laughing at my Nightmare. Spinal muscular atrophy has kept him in a wheelchair since he was 2 years old, preventing him from keeping his muscles. On the blog, Burcaw, 25, shares amusing observations, happy moments and painful experiences with half a million followers. That has led to book deals, speaking engagements and a nonprofit organization that helps people with muscle diseases buy equipment. In late June, the call Burcaw was waiting for came. He found out his insurance will cover the first drug approved by the government to treat his disease. Spinraza, which costs $750,000 the first year, and $375,000 every year after that. His first drug injection, which he received a couple of weeks ago, is expected to help him maintain, and maybe even gain, strength. He said his second would occur around Labor Day, a holiday many associate with efforts … Continue reading →

This year, collecting donations to go toward research for treating muscular dystrophy during the Lynchburg Fire Departments Fill the Boot campaign has a different meaning for David Cox. Cox had worked for the department for more than 15 years but retired at the beginning of August after he was diagnosed with Myasthenia Gravis, a neuromuscular disease, at the end of May. Like other muscular dystrophy-related diseases, Myasthenia Gravis weakens muscles as an autoimmune disease that damages connections between nerves and muscles, according to the Muscular Dystrophy Association. Hed participated in the departments fundraisers for the Muscular Dystrophy Association for years and decided to come back post-retirement to help out again this year. It really does mean more now, you know, he said. Out here today, my legs were shaking this morning I was like, I dont know if Im going to do it, but I said, Why not? Its for a good cause. I can suck it up for three or four hours and just do it. Some days, Cox said he can only take a few steps before becoming exhausted and needing to rest 10 to 15 minutes, whereas he was able to exercise normally before starting to get … 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 →