Category Archives: Muscular Dystrophy Treatment

A petition for the Federal Drug Administration to consider a new drug treatment for Duchenne muscular dystrophy is close to a critical milestone. WLWT News 5 spoke with a Cincinnati family Monday whose 3-year-old son, Mason, has Duchenne muscular dystrophy. The Diersing Family wanted to raise awareness about the disease and a new treatment that is up for consideration. They asked people to sign an online petition for the FDA to consider a new experimental drug for treating the genetic disease. I tell people that it's a mourning period that you go through. You really mourn a life that you thought was going to be one way and it's going to be drastically different, said Katherine Mayrhofer Diersing, Masons mother. The petition is only 3,000 signatures from 100,000. If the petition reaches 100,000 signatures by Saturday, the White House said it will take action on the proposal. We have no time. Science is finally on our side. We have no time and the Food and Drug Administration is dragging its heels, said Dr. Benjamin Seckler, a physician who founded the national nonprofit organization fighting for DMD Charleys Fund said. Children are dying. To sign the petition or find out more … Continue reading →

Winter Storm Watchissued March 25 at 3:47PM MDT expiring March 28 at 6:00AM MDT in effect for: Chaffee, Conejos, Lake, Mineral, Rio Grande, Saguache Winter Storm Watchissued March 25 at 4:10PM MDT expiring March 28 at 9:00AM MDT in effect for: Eagle, Garfield, Moffat, Rio Blanco, Routt Winter Storm Watchissued March 25 at 4:10PM MDT expiring March 28 at 9:00AM MDT in effect for: Archuleta, Delta, Dolores, Eagle, Garfield, Gunnison, Hinsdale, La Plata, Mesa, Montezuma, Montrose, Ouray, Pitkin, San Juan, San Miguel Winter Storm Watchissued March 25 at 3:59PM MDT expiring March 28 at 9:00AM MDT in effect for: Eagle, Garfield, Moffat, Rio Blanco, Routt Winter Storm Watchissued March 25 at 3:59PM MDT expiring March 28 at 9:00AM MDT in effect for: Archuleta, Delta, Dolores, Eagle, Garfield, Gunnison, Hinsdale, La Plata, Mesa, Montezuma, Montrose, Ouray, Pitkin, San Juan, San Miguel Fire Weather Warningissued March 25 at 3:08PM MDT expiring March 26 at 8:00PM MDT in effect for: Baca, Bent, Crowley, El Paso, Fremont, Huerfano, Kiowa, Las Animas, Otero, Prowers, Pueblo Fire Weather Watchissued March 25 at 4:13AM MDT expiring March 26 at 8:00PM MDT in effect for: Baca, Bent, Crowley, El Paso, Fremont, Huerfano, Kiowa, Las Animas, Otero, Prowers, Pueblo … Continue reading →

Get Breaking News First Receive News, Politics, and Entertainment Headlines Each Morning. SACRAMENTO (CBS13) A family is fighting for the life of their young son. He had been undergoing treatment, getting an experimental drug through a study at the UC Davis Medical Center. But that study has ended, and now they fear that he could lose his battle. Ryan Dunne goes through the motions other 9-year-olds take for granted. He struggles, his muscles failing him more and more. He has Duchenne Muscular Dystrophy; his muscles are wasting away. Things are tough, he has a hard time getting up and down stairs, said Ryans mother, Jennifer Dunne. As his muscles give out, its a near certainty the disease will rob Ryan of his mobility, and his life before hes 30. Its a progressive disease. Every day he gets weaker, Jennifer said. There was a time when the downward spiral slowed: When Ryans family was flying weekly from Colorado to Sacramento, taking part in a clinical trial with the UC Davis Health System. DMD is actually is the most common form of muscular dystrophy in children. Its actually due to absence of one of the most common and ubiquitous proteins in the … Continue reading →

Lexington, MA (PRWEB) March 24, 2014 Today, the FSH Society, a Massachusetts based non-profit that is a world leader in combating facioscapulohumeral dystrophy (FSHD), announced that it has awarded six grants totaling more $609,525 to new research projects. Through these studies, the FSH Societys fellowship program aims to gain insights and achieve significant milestones into the research of FSHD, one of the most prevalent types of muscular dystrophy. A degenerative muscle disease, FSHD causes progressive weakness, usually starting with the face, shoulder and arms, but can affect almost any skeletal muscle. FSHD affects approximately 500,000 people worldwide and between one and two percent of the population carries a genetic trait that places future generations at risk of the disease. Currently, there is no cure or effective treatment. Research grants most recently awarded by the FSH Society include: 1.Investigating effects of PARP1 inhibitors in DUX4 expression ($89,267) Yi-Wen Chen, D.V.M., Ph.D. George Washington University and Childrens National Medical Center (Washington, D.C.) A mysterious protein called DUX4 is believed to cause FSHD. The findings of the study will provide insights of the involvement of PARP1, a promoter of the DUX4 gene, in FSHD, and will have a direct impact on developing therapeutics … Continue reading →

North Royalton, OH (PRWEB) March 21, 2014 John Owen's Adventure Inc. is thrilled to launch the second annual Play for JOA-Dodgeball event bringing together children and adults alike to enjoy this classic sport while fundraising to cure the fatal disease Duchenne Muscular Dystrophy (DMD). "Our 2013 Tourney was a huge success," said Jen Dumm, Vice President of John Owen's Adventure Inc. (JOA). "Last year brownie troops, soccer and baseball teams participated in dodgeball games, won prizes and had a whole lot of fun. We plan to make this year's round two even better!!!" Play for JOA-Dodgeball can serve as a bonding experience as well as a community service project for sports team, families or friends. Join today for only $5 per person and enjoy running around with friends and meeting new ones. The Play for JOA-Dodgeball event will take place on Saturday, March 29th from 4-7 p.m. at the North Royalton Fieldhouse located at 10701 Royalton Rd in North Royalton. For questions and information contact Amy Schultz at 440-230-9486 or visit the JOA events page at http://www.joainc.org. Duchenne Muscular Dystrophy (Duchenne) is a fatal progressive disorder that causes loss of muscle function and independence, affecting mainly young boys. John Owens … Continue reading →

OILDALE, CA - It is a disease that has no known cure. Although advances are being made in the treatment of Muscular Dystrophy, an Oildale family says it can't come soon enough. The Montero family learned just two months ago, their 5-year-old son has Duchenne Muscular Dystrophy. They've been researching treatment options and came across a drug called Eteplirsen. But, it is currently in the trial phase with the FDA. Five-year-old Anthony Montero is all smiles as he plays with his sister and family. You wouldn't know he has Muscular Dystrophy, a disease that attacks the muscle fibers. "When he had Muscular Dystrophy it was the hardest. That is when we started looking up the types of Muscular Dystrophy and Duchenne is the one that came up," said Maria Montero, Anthony's mother. Doctors say most children with Duchenne will need a wheelchair before they reach their teens and often die from the disease in their twenties. "My son talks about it every day. He wants to be a firefighter or a police officer, and only I know the truth if he can't get this medicine," added Maria. Determined to do whatever they can, the Monteros came across information regarding a … Continue reading →

Parent Project Muscular Dystrophy logo. (PRNewsFoto/Parent Project Muscular Dystrophy) HACKENSACK, N.J., March 20, 2014 /PRNewswire-USNewswire/ -- Nationwide Children's Hospital was named the first Certified Duchenne Care Center by Parent Project Muscular Dystrophy (PPMD), the leading advocacy organization working to end Duchenne muscular dystrophy (Duchenne). PPMD is launching the Certified Duchenne Care Center Program as part of its robust Transforming Duchenne Care Initiative. Over the coming year, plans are being made to award other qualified centers the title of Certified Duchenne Care Center in an effort to ensure centers maintain the highest standards in clinical and sub-specialty services, rapidly apply new evidence based knowledge, and comply with standards in clinical care that were established by the CDC Care Considerations. As part of its ongoing mission to end Duchenne, PPMD promises to insist that all people with Duchenne receive comprehensive care. PPMD Founding President and CEO, Patricia Furlong, explained the reason for the new certification program: "There are 10-12,000 young men (and some women) in the U.S. living with Duchenne muscular dystrophy. Every one of these people deserves the best care and treatment possible. And while each of them may receive care from a local or in many cases, not so local … Continue reading →

By Jennifer Jarrell Thursday March 20, 2014 11:37 PM UPDATED: Friday March 21, 2014 12:48 AM COLUMBUS, Ohio - It's a deadly disease without a cure, but researchers say they have a drug that slows the progression. Now, families of boys with a rare form of muscular dystrophy are pushing for government approval. 7-year-old Jackson Maynard can play football with his big brother right now, but he likely won't be able to in a few years. "He's starting to fall a lot at school, and he has difficulty getting up and down the steps," Jackson's mother, Kelly Maynard, said. Maynard said her son was diagnosed with Duchenne Muscular Dystrophy two years ago. She said her little boy is losing strength every day. "Boys are usually in a wheelchair by the time they are 10 or 12. It is a 100% fatal disease, most boys don't live past their 20s with this disease," Maynard said. Researchers, including a doctor from Nationwide Children's Hospital in Columbus, recently went to Washington to testify on behalf of the drug Eteplirsen. Dr. Jerry Mendell said the drug works. Maynard isn't able to give the drug to her son because it still hasn't been approved by … Continue reading →

Get CBS4 News Updates In Your Inbox Sign up for News, Sports, Broncos and Health Emails. DENVER (CBS4) With passion and tears Jennifer Dunne pleads for answers and action from the Food and Drug Administration, which has held up the use of an experimental drug that has shown promise for children like her son, a 9-year-old with Duchenne Muscular Dystrophy, a 100 percent fatal disease. They need to move we have no more time, no more time, Dunne told CBS4. Her son, Ryan, a third-grader, was diagnosed with DMD years ago. It is a genetic, muscle-wasting disease that hits young boys and is always fatal, usually by the time the patient reaches their mid-20s. But two experimental medications have shown great promise in treating the disease. In 2013, Ryan was placed on one of the medications as part of a six-month trial study. When there was no hope, all of a sudden things were getting better, said Ryans father, Chris. He walked further, had better stamina and energy and didnt fall into bed saying, Im tired, said his mother. And when he was pulled off of it he went downhill immediately. The drug is effective. A second medication that has … Continue reading →

(Nasdaq:RNA), the Dutch biopharmaceutical companyfocusing on RNA-modulating therapeutics for rare diseases with high unmet need,today reported financial results for the full year ending December 31, 2013,and provided an update on the next steps for its exon-skipping platform for thetreatment of Duchenne Muscular Dystrophy (DMD). 'As we have previously stated, we are encouraged by initial findings fromfurther analyses of the aggregate drisapersen data that suggest that treatingearlier in DMD and treating longer shows a delay in the progression of thedisease. These data give us the confidence to engage patient groups, clinicalexperts and regulators to explore a path forward for drisapersen,' said HansSchikan, CEO of Prosensa. Mr. Schikan continued, 'While the past few months have been a very importanttransition period for Prosensa, our primary focus has not changed, which is toimprove the lives and outcomes of boys with DMD. At the start of the year weregained the rights to drisapersen and retained rights to our otherdevelopment-stage compounds for DMD from GlaxoSmithKline (GSK). We arecurrently working closely with GSK to transfer data and other elements coveredin the collaboration as soon as practicable and within 120 days of the January12 effective date of the agreement. Concurrently, we are focused on thecontinued analysis of the … Continue reading →