Category Archives: Muscular Dystrophy Treatment

In 1965, a coach for the University of Florida football team the Gators noticed that his players were being affected by heat-related illnesses. The coach then asked a team of physicians to determine the exact cause for his players falling ill. The physicians discovered that the team was losing more fluids than they could replenish. Thats when Gatorade was invented. Why is Gatorade better than water? Gatorade contains electrolytes. Electrolytes are chemicals such as sodium, magnesium, calcium, potassium and other key nutrients that aid in the function and overall health of our cells. They help improve hydration by directing water to the parts of the body where its needed and by maintaining fluid balance inside the cells. Electrolytes get their name from the act of sending electrical charges to muscles to help them move and contract. (This is true of Gatorade and other brands similar to it.) Ive been criticized for drinking Gatorade. They say it has too much salt and sodium for someone who isnt active. But for a person like me, whose muscles and cells dont function properly, the electrolytes in Gatorade help keep my heart rate steady and my body hydrated. I do, however, drink the G2 … Continue reading →

By Ed Stannard, estannard@nhregister.com @EdStannardNHR on Twitter NEW HAVEN >> Yale University allegedly has the fourth-worst record of mistreatment of rats and mice used in research, according to a report from People for the Ethical Treatment of Animals. On Monday, PETA reported its findings of animal-welfare violations at the top 20 universities that receive grants from the National Institutes of Health. The results were presented at the Tenth World Congress on Alternatives and Animal Use in the Life Sciences conference in Seattle. Through Freedom of Information Act requests, PETA found that between Jan. 1, 2015, and April 1, 2017, there were 430 violations of agreements each of the 20 universities has with the Public Health Service. Those agreements are a condition of receiving NIH grants. Yale had 39 violations, almost twice the average of 21.5 which means Yale is doing significantly more poorly than average, according to Alka Chandna, PETAs chief of laboratory case management. Yale received $314 million in NIH grants in 2016, PETA said. Among the alleged violations at Yale, according to PETA: A mouse rack with 141 cages was disconnected from the automatic watering system, resulting in nine deaths and 93 other mice showing sever signs of … Continue reading →

LOS ANGELES Jerry Lewis, whose career as producer-director-writer and movie star peaked in the 1960s, may be best remembered by younger generations for the muscular dystrophy telethons he conducted every Labor Day weekend. While he had done earlier fundraising specials, Lewis appeared in his first Labor Day telethon, broadcast on a single New York station, in 1966. Donations totaled $1,002,114. He continued each year as the telethon grew into a huge event, seen on more than 200 stations. By 2007, the yearly take was nearly $64 million, and the Muscular Dystrophy Association said the telecast had raised $1.46 billion to fight the disease since it began. Comedian Jerry Lewis during an interview at the Four Seasons Hotel in Los Angeles, on Aug. 24, 2016. Rich Fury / Rich Fury/Invision/AP Muscular dystrophy is a group of genetic diseases characterized by progressive weakness and degeneration of muscles that control movement. The most severe and most common childhood form is Duchenne muscular dystrophy, which occurs in about one in every 3,500 male births. Lewis died Sunday in Las Vegas. He was 91. "MDA would not be the organization it is today if it were not for Jerry's tireless efforts on behalf of 'his … Continue reading →

While there is no cure for Friedreichs ataxia, there are treatments thatcan help patients manage symptoms and improve their quality of life.Treatment for Friedreichs ataxia consists of medications, physicaltherapy, and surgery according to the Muscular Dystrophy Association. MORE:Five major symptoms of Friedreichs ataxia Heart problems associated with Friedreichs ataxia can be managed with drugs, includingdiuretics,ACE inhibitors, andbeta blockers which can all be used to lower the hearts workload. Insulin is used for those who suffer from ataxia-related diabetes. Physical therapy is an important part of treatment for Friedreichs ataxia, and although it wont stop muscle weakness it can help to enhance flexibility. For those who suffer from speech problems or difficulty eating, speech therapists can devise special exercises to help improve these areas. Surgical procedures can help correct some of the skeletal problems associated with the condition. Scoliosis surgery can help straighten the back for people who suffer from curvature of the spine and there are surgical procedures to address foot deformities. Research into the role of frataxin in Friedreichs ataxia is looking at new possible treatments that mayfix the genetic mutation so as to slow down or reverse the course of the disease. MORE: Five types of treatment for … Continue reading →

COLORADO SPRINGS, Colo. Local firefighters are showing their support for the Muscular Dystrophy Association (MDA) as they kick off the annual Fill the Boot fundraising campaign to help kids and adults with muscular dystrophy, ALS and related muscle-debilitating diseases live longer and grow stronger. From August 24 to September 4, firefighters from Colorado Springs Fire Department and the International Association of Fire Fighters (IAFF) Local #5 as well as several other fire departments in southern Colorado will hit storefronts with boots in hand asking pedestrians, drivers, customers and other passersby at various locations in Colorado Springs, Pueblo and more southern Colorado towns to make a donation to MDA. This year, Local #5, in collaboration with MDA and Papa Johns, will host a pizza party for CSFD Fire Chief Ted Collas, members of Colorado Springs City Council, local families served by MDA and southern Colorado firefighters. The pizza party will celebrate the breakthrough of four new drug treatments for muscle disease in the past 11 months and will take place at Colorado Springs Station 19 on August 22 at 11:30 a.m. Funds raised through Fill the Boot also help finance research and life-enhancing programs such as state-of-the-art support groups and Care … Continue reading →

Cardiac stem cell infusions could someday help reverse the aging process in the human heart, making older ones behave younger, according to a new study from the Cedars-Sinai Heart Institute. "Our previous lab studies and human clinical trials have shown promise in treating heart failure using cardiac stem cell infusions," said Eduardo Marbn, MD, PhD, director of the Cedars-Sinai Heart Institute and the primary investigator of the study. "Now we find that these specialized stem cells could turn out to reverse problems associated with aging of the heart." In the study, investigators injected cardiosphere-derived cells, a specific type of stem cell known as CDCs, from newborn laboratory rats into the hearts of rats with an average age of 22 months, which is considered aged. Other laboratory rats from the same age group were assigned to receive placebo treatment, saline injections instead of stem cells. Both groups of aged rats were compared to a group of young rats with an average age of 4 months. Baseline heart function was measured in all rats, using echocardiograms, treadmill stress tests and blood analysis. The older rats underwent an additional round of testing one month after receiving cardiosphere-derived cells that came from young rats. … Continue reading →

Tuesday, August 15, 2017 8:13 a.m. CDT by Jon DeMaster SHEBOYGAN, Wis. (WHBL) - The Sheboygan Fire Department Local 483 is showing its support for the Muscular Dystrophy Association as its members kick off the annual Fill the Boot fundraising campaign to help kids and adults with muscular dystrophy, ALS and related muscle-debilitating diseases live longer and grow stronger. It will take place August 17th and 18th. Continuing a more than 60 year tradition, twenty dedicated fire fighters from Sheboygan Fire Department Local 483 will hit the streets with boots in hand asking pedestrians, motorists, customers and other passersby to make a donation to MDA, they will be at the intersection of 25th and Superior and 8th Street and Erie between 11am 5:00pm both days. We are thrilled to be working with the Sheboygan Fire Department Local 483 for another year of Fill the Boot 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 Fundraising Coordinator Murphy Maes. The dedication of these fire fighters to MDAs mission is unwavering, spending countless hours both with Fill the Boot and MDA Summer Camp to care for … Continue reading →

Cardiff Universityscientists, partneringwith the biotech company PerkinElmer, have developed a diagnostic kit to screen for Duchenne muscular dystrophy (DMD) in newborns using dried blood spots, according to researchers. The study, Characterization of a Blood Spot Creatine Kinase Skeletal Muscle Isoform Immunoassay for High-Throughput Newborn Screening of Duchenne Muscular Dystrophy, was published in the journal Clinical Chemistry. This new diagnostic method works by detecting the presence of the enzyme CK-MM, which is elevated in patients with DMD due to muscle damage. The test was created by Dr. Ian Weeks, a professor at Cardiff Universitys School of Medicinein Wales and the lead author of the study. The test was then adapted into an existing assay developed by PerkinElmer. The successful outcome of this study is an example of the impact that can be achieved through collaboration between academic, clinical and industry colleagues in developing improvements in human healthcare, Weeks said in a press release. Previous attempts at developing a diagnostic kit using blood spot CK (creatinine kinase) enzyme assays were met with little success because the tests were nonspecific and the results were unreliable. This is due to the fact that prior tests could not distinguish between the three different forms of … Continue reading →

The investigational therapy MA-0211, also called MTB-1, a potential treatment for Duchenne Muscular Dystrophy (DMD), was approved for a Phase I clinical trial, developerMitobridge announced. The trial will evaluate the therapys safety and tolerability and determine its pharmacokinetics and pharmacodynamics profile, or its behavior in the body and mechanism of action, in healthy volunteers. The data collected will then be used to launch a trial with DMD patients. Mitobridgeis a company focusing on new small molecule therapeutics to improve mitochondrial functions. MA-0211, a modulator of peroxisome proliferator-activated receptor (PPAR) delta, was developed under the companys mitochondrial enhancement platform in collaboration with corporate partnerAstellas Pharma. It is the platforms first therapy being tested in a clinical study. The trial was established after extensive preclinical studies, either with DMD patient samples or animalmodels for the disease, which showed MA-0211 has therapeutic benefits. DMD patients exhibit mitochondrial defects that contribute to the diseases progression. Treatment of patient muscle cells with MA-0211 increased the expression of numerous genes involved in a key metabolic pathway known as fatty acid oxidation that occurs in mitochondria and is vital for producing the energy cells need to function properly. The treatment increased the number of mitochondria and improved … Continue reading →

INDIANAPOLIS Jordan McLinn is an eight-year-old boy with a disease with no known cure. His condition, Duchenne muscular dystrophy, is caused by a genetic mutation that prevents the body form producing a protein that the muscles need to work properly called dystrophin. Without the protein, the muscles become damaged and weak. The disease hinders the ability to walk and breath, and eventually becomes fatal. When we first started lobbying for the right to try in Indiana, Jordan did not qualify for clinical trials, Laura McLinn, Jordans mom, said. There was this drug that existed, kind of just dangling in front of our eyes, and my son did not qualify for a clinical trial. The McLinn family helped get the Right to Try legislation unanimously passed in Indiana in 2015. Now they are working with U.S. Sen. Joe Donnelly to get similar legislation, which allows families to exhaust all treatment options for a terminally ill family member, passed nationally. Donnellys Right to Try Act gives families in every state who do not qualify for clinical trial, the ability to try drugs that have not been approved, but have passed the first stage of the Food and Drug Administration testing. Donnelly was … Continue reading →