Category Archives: Muscular Dystrophy Treatment

Public release date: 27-Mar-2013 [ | E-mail | Share ] Contact: Tanya Gubbay tanya.gubbay@rhul.ac.uk Royal Holloway, University of London Royal Holloway, is today (Wednesday) giving money generated by its research into Duchenne muscular dystrophy to the Muscular Dystrophy Campaign. Families affected by the condition are also visiting laboratories on campus to find out from the scientists about new drug developments and see the work being carried out. Affecting just 1 in 3000 young boys, Duchenne muscular dystrophy is a life-shortening condition, which causes muscles to weaken and waste over time leading to increasingly severe disability. Professor George Dickson from the School of Biological Sciences at Royal Holloway has been leading a team of scientists looking into pioneering treatments, including exon skipping, a process that looks to encourage cellular machinery to 'skip over' an exon which makes up part of the gene. It is thought that by skipping one or two exons, it may be possible to treat around 83% of the genetic errors causing Duchenne muscular dystrophy. A pharmaceutical company is now looking to collaborate with the team to develop drugs that could be used for treating the condition and has paid a fee to secure rights to the research. … Continue reading →

By Terry Chrisomalis - March 25, 2013 | Tickers: BMY, PFE, SRPT | 0 Comments Terry is a member of The Motley Fool Blog Network -- entries represent the personal opinion of the blogger and are not formally edited. There is currently a big medical need for a drug that treats Duchenne Muscular Dystrophy. This disease basically halts motor function and movement as the years go bye. There are hardly any options for these patients, and a breakthrough drug from a pharmaceutical company can prove to be monumental for them. One such company has achieved tremendous results in Duschenne Muscular Dystrophy, and this company is known as Sarepta Therapeutics(NASDAQ: SRPT). Results that matter On Oct. 3, 2012, Sarepta Therapeutics announced results from its phase two trial in Duschenne Muscular Dystrophy. Patients in the study, who took eteplirsen, were on average able to walk 89 meters more than those who just took the placebo drug instead after 48 weeks. What's even more incredible is that when patients had muscle biopsies performed on them at the beginning of the study, researchers noticed that they had no dystrophin proteins being created. But by the end of the study, the levels of dystrophin protein … Continue reading →

CARLSBAD, Calif., March 20, 2013 /PRNewswire/ --Isis Pharmaceuticals, Inc. (ISIS) announced that data from the Phase 1 study of ISIS-SMNRx in children with spinal muscular atrophy (SMA) were presented today at the 65th American Academy of Neurology (AAN) Annual Meeting by Dr. Claudia Chiriboga, from Columbia University Medical Center. In the presentation, Dr. Chiriboga reported that, in this single-dose, open-label study, ISIS-SMNRx was well tolerated in children with SMA at all dose levels tested and that improvements were observed in Hammersmith scores, a measure of muscle function, in a number of the children. SMA is a severe and rare genetic neuromuscular disease characterized by muscle atrophy and weakness and is the most common genetic cause of infant mortality. ISIS-SMNRx is an antisense drug designed to treat all types of SMA. "SMA is a devastating disease that results in severe muscle weakness and respiratory failure in infants and children. Current treatments are supportive and do not address the underlying genetic cause of SMA, the loss of SMN protein, which is critical to the health and survival of nerve cells responsible for neuromuscular growth and function. ISIS-SMNRx is designed to address the underlying genetic problem for patients with SMA by increasing the … Continue reading →

Get your name down for 'Town and Gown' 6:00pm Tuesday 19th March 2013 in News By Francesca Bardsley A MAN with a neuromuscular condition has urged runners to sign up for a race which raises cash for vital medical research. Management consultant Roger Wild, 62, has spinal muscular atrophy, which causes severe muscle weaknesses. He said the Town and Gown 10km run supported by the Oxford Mail on Sunday, May 12, provides much needed funds for the Muscular Dystrophy Campaign. He said: It is incredibly important to do the Town and Gown, particularly in a place like Oxford. Im sure academics and the citys population can really get to grips with the science of eradicating an incredibly debilitating condition, for which the Muscular Dystrophy Campaign is a driving force. The more we can do with cell research and treatments and the more people can run and raise money, the better. Mr Wild, who uses a powered wheelchair, has campaigned for better treatment for neuromuscular conditions. He is regularly invited to contribute to an all-party parliamentary group on the disease. Treatment varies depending on the priority given the conditions by 10 NHS strategic health authorities, which cover English regions, he said. … Continue reading →

Thirteen-year-old Austin and 10-year-old Max Laclaire play together with their Ben 10 action figures in Saxtons River. (Josh Stilts/Reformer file photo) BRATTLEBORO -- Laurie Webb wanted to put on a benefit for her grandsons with Duchenne Muscular Dystrophy. And, fortunately, she knows plenty of musicians. Webb was able to recruit three area bands -- Wyld Nightz, Pretty Daddy and Otis and the Elevators -- to perform at an event scheduled for Saturday at the American Legion. "Dancing for Duchenne" is set to begin at 7 p.m. and raise money for a trio of charities dedicated to helping those affected by DMD. Webb's daughter, Jenn McNary, has six children, two of whom suffer from Duchenne, an ultimately fatal degenerative muscle disease that affects roughly 20,000 children in the United States. Both Austin and Max Leclaire use the support of their family to live independent and productive lives, though their rare condition makes it a challenge. Max, 11, was eligible for a medical trial of Eteplirsen, a drug that greatly improved his quality of life. But Austin, 14, did not qualify and his condition has gotten progressively worse without the treatment. Max is able to walk while his older brother gets around … Continue reading →

CAMBRIDGE, Mass., March 20, 2013 /PRNewswire/ --DART Therapeutics Inc., an innovative, new-model biotechnology firm focused on developing therapies for Duchenne muscular dystrophy (DMD), announced today that it is developing a SARM drug candidate obtained from Belgium-based Galapagos NV. In early studies, the drug candidate, renamed DT-200, demonstrated significant potential to increase muscle size and strength. DT-200 could represent a new class of therapy for DMD, a muscle-wasting disease, and offer potential benefit for multiple neuromuscular diseases where improved muscle strength and function would be beneficial. Galapagos has provided the rights for its SARM drug candidate in DMD to the patient foundations Charley's Fund and theNash Avery Foundation, who co-founded DART Therapeutics. Terms of the rights transfer from Charley's Fund and Nash Avery to DART were not disclosed. Selective androgen receptor modulators--or SARMs--promote increased muscle mass and thereby strength--through normal androgenic pathways without the negative effects of oral androgenic steroids. DT-200 is an orally available SARM that has demonstrated potential to increase muscle size and strength in preclinical studies. In DMD, SARMs could be useful in halting progressive muscle loss by increasing the size and strength of diseased muscle. However, this remains an unexplored area of DMD therapy. Although the drug … Continue reading →

HACKENSACK, N.J., March 21, 2013 /PRNewswire-USNewswire/ --Parent Project Muscular Dystrophy (PPMD) has awarded University of Minnesota researcher Dr. Rita Perlingeiro a grant for $220,000 to develop a therapeutic approach for Duchenne muscular dystrophy (Duchenne) using a new type of "adult-derived" stem cell called "induced pluripotent stem cells" or "iPSCs." iPSCs can be created from non-controversial sources like skin, have the ability to multiply indefinitely, and can be coaxed to become many different kinds of tissue types. (Logo: http://photos.prnewswire.com/prnh/20100119/DC39975LOGO) Previously, Dr. Perlingeiro's group demonstrated that skin cells from donor mice that lack dystrophin could be converted into iPSCs, and then converted again into muscle stem cells with the mutation corrected using a modified viral carrier. When these corrected mouse stem cells were transplanted into recipient mice that lacked dystrophin, the mice showed improved muscle strength. Now Dr. Perlingeiro will repeat this process with funding from PPMD, this time using human skin cells from donors with Duchenne and using a safer method of correcting the dystrophin deficiency that does not rely upon random integration. The goal is to start laying the groundwork for an FDA-approved stem cell treatment for Duchenne. "Translating results in mice to treatments in humans is never straightforward," said … Continue reading →

To view our videos, you need to enable JavaScript. Learn how. install Adobe Flash 9 or above. Install now. Then come back here and refresh the page. Eight-year-old Alesky Magno was born with muscular dystrophy. Four years ago, he contracted a dangerous intestinal infection caused by the clostridium difficile bacteria, also known as C-diff. "Antibiotics weren't working for him," Guillermina Magno, Alesky's mother and a donor of a fecal transplant patient, said through an interpreter. "They were producing changes in his daily life. And every time he would eat, he would get diarrhea." Magno's doctor at New York Presbyterian/Morgan Stanley Children's Hospital suggested a treatment rarely done on children: transplanting a healthy person's feces, full of good bacteria, into the infected colon. It was a risk that the Magnos say was worth taking. "After the transplant, his daily life got much better," Paz Magno, Aleksy's father, said through an interpreter. "Now, he doesn't have pain." A recent New England Journal of Medicine Study found that fecal transplants are a significantly more effective treatment for C-diff than the antibiotic, vancomycin. The study, though, focused on adults with the infection. There's still little research done with children, which is why pediatric gastroenterologist … Continue reading →

Duchenne Muscular Dystrophy Treatment and Symptoms XXXXXX◅ Duchenne Muscular Dystrophy treatment mdash; Finding the right information about Duchenne Muscular Dystrophy treatment symptoms, is crucial to managing... By: MainMDcom … Continue reading →

AMBITION FOR MD Ambition for Defying Muscular Dystrophy. By: DefyingDystrophy … Continue reading →