Category Archives: Muscular Dystrophy Treatment

GOSHEN Lisa Crawford wasnt looking for a handout when she stopped by the College Avenue Fire Station last February. She wanted to let the firemen know about the upcoming Muscular Dystrophy Association (MDA) Muscle Walk and hoped they might donate a little to help find a cure. Ever since her 9-year-old son Brendon was diagnosed with Duchenne Muscular Dystrophy, a 100 percent fatal disease, Lisa has been on a quest to see that a cure is found before she loses her son. This severe, progressive muscle-wasting condition causes most children to be wheelchair bound by the time they are 12. Life expectancy is late teens to early 20s. What Lisa got when she entered that fire station was not only money to donate to the MDA Muscle Walk, but financial assistance for her family and a friendship between firemen and family that will stay the course. She didnt ask us for help, said Fireman Courtney Snyder. But we were looking for someone in the community who we could support in some way. Snyder got the OK from the Goshen Professional Fire Fighters Local 1443 and the station has helped the family with its frequent trips to Cincinnati for doctors visits. … Continue reading →

SAXTONS RIVER, Vt. Jenn and Craig McNary and their family of six are in a tough spot. Their two oldest sons have Duchenne Muscular Dystrophy, a rare form of muscular dystrophy that only affects boys. However, only one son, Max, 10, was chosen to be in an experimental drug trial that seems to be reversing his symptoms. About 16 weeks into treatment, Max started showing signs that he was building strength. While his condition was improving on the drug eteplirsen, however, his older brother Austin, 13, was watching from his wheel-chair. "Around week 36, we started telling people, 'Hey, this drug is working. Now we need it for Austin and all the other boys, what's the fastest route for this?'" said Jenn McNary. As the parents soon found out, there really is no fast route. The family has drafted a petition to the Food and Drug Administration, hoping that the agency would speed up the drug's approval process through a new orphan drug bill, passed by Congress this summer. "The FDA has its red tape," said Craig McNary, "and the law says they'll do things faster, but how fast is fast?" For over seven months, the family has been trying … Continue reading →

Recent scientific breakthroughs are offering real hope for patients living with one of the most common forms of muscular dystrophy. Scientists from around the world converged on San Francisco for the FSH Society's International Research Consortium meeting to share the latest advances in facioscapulohumeral muscular dystrophy (FSHD) research, including a new genetic explanation for the muscle-wasting disease. San Francisco, CA (PRWEB) November 08, 2012 Thanks to the efforts of Perez and many other patients, FSHD research has come a long way. The evidence was there for all to see at the FSH Societys 2012 International Research Consortium & Research Planning Meeting, held on November 6, 2012, in conjunction with the annual meeting of the American Society of Human Genetics in San Francisco, CA. The biggest buzz at the meeting was generated by the discovery of a new gene for FSHD by scientists from the U.S., Netherlands and France. The study by Richard Lemmers and colleagues will be published in Nature Genetics on November 11. Over 90 percent of FSHD patients have a shortening of the D4Z4 region on chromosome 4, which results in the expression of a gene called DUX4, with apparent toxic effects in muscle. The newly found gene … Continue reading →

Public release date: 7-Nov-2012 [ | E-mail | Share ] Contact: Sally Stewart Sally.stewart@cshs.org 310-248-6566 Cedars-Sinai Medical Center LOS ANGELES Nov. 7, 2012 Cedars-Sinai Heart Institute researchers have found that an experimental compound may help stem the debilitating effects of muscular dystrophy by restoring normal blood flow to muscles affected by the genetic disorder. The researchers studied HCT 1026, a new type of molecule in which nitric oxide is chemically attached to a standard anti-inflammatory agent, in a preclinical model of muscular dystrophy. Results suggest HCT 1026 may be beneficial for the treatment of Duchenne muscular dystrophy, which begins in early childhood, and Becker muscular dystrophy, which often occurs later in adulthood. Preliminary results were presented in April 2012 at the experimental Biology meeting and now the full study is published in the Public Library of Science (PLOS ONE) and is available online. Both forms of muscular dystrophy are caused by problems with a protein called dystrophin, which helps maintain healthy muscles. If patients have less dystrophin protein or if their body manufactures dystrophin protein that does not function correctly, their muscles cannot work properly and eventually become permanently damaged. As diseased muscles weaken over time, patients gradually can lose … Continue reading →

ScienceDaily (Nov. 7, 2012) Cedars-Sinai Heart Institute researchers have found that an experimental compound may help stem the debilitating effects of muscular dystrophy by restoring normal blood flow to muscles affected by the genetic disorder. The researchers studied HCT 1026, a new type of molecule in which nitric oxide is chemically attached to a standard anti-inflammatory agent, in a preclinical model of muscular dystrophy. Results suggest HCT 1026 may be beneficial for the treatment of Duchenne muscular dystrophy, which begins in early childhood, and Becker muscular dystrophy, which often occurs later in adulthood. Preliminary results were presented in April 2012 at the experimental Biology meeting and now the full study is published in PLoSONE and is available online. Both forms of muscular dystrophy are caused by problems with a protein called dystrophin, which helps maintain healthy muscles. If patients have less dystrophin protein or if their body manufactures dystrophin protein that does not function correctly, their muscles cannot work properly and eventually become permanently damaged. As diseased muscles weaken over time, patients gradually can lose their ability to walk, sit or use their muscles in other ways. There is no cure now for either form of the disease. "There is … Continue reading →

BOSTON (TheStreet) -- Duchenne muscular dystrophy (DMD) advocacy groups are mobilizing to support efforts by Sarepta Therapeutics (SRPT) to seek accelerated approval for its experimental drug eteplirsen. Some of these groups, accompanied by parents of DMD patients, have already met with U.S. Food and Drug Administration officials to make sure regulators understand the urgent need for new therapies. More meetings with U.S. regulators are planned. Sarepta will meet with the FDA, likely early next year, to present results from the eteplirsen phase IIb study and request permission to file for accelerated approval. The company is holding a conference call Wednesday in conjunction with third-quarter financial results. See if (SRPT) is in our portfolio "We are absolutely going to do everything we can to get eteplirsen approved," said Sharon Hesterlee, senior director of research at Parent Project Muscular Dystrophy (PPMD), the largest DMD non-profit in the U.S. Originally posted here: Advocates, Patients Rally To Support Sarepta Early Drug Approval Push … Continue reading →

Dr. William Rader - Breakthrough Stem Cell Therpay for Muscular Dystrophy http://www.drwilliamrader.com - New Stem Cell Therpay for Muscular DystrophyFrom:William RaderViews:5 0ratingsTime:06:00More inScience Technology Continue reading here: Dr. William Rader - Breakthrough Stem Cell Therpay for Muscular Dystrophy - Video … Continue reading →

William Rader MD - Breakthrough Muscular Dystrophy Treatment http://www.drwilliamrader.com - Stem Cell Treatment for Muscular DystrophyFrom:William RaderViews:5 0ratingsTime:01:57More inScience Technology Read this article: William Rader MD - Breakthrough Muscular Dystrophy Treatment - Video … Continue reading →

(Corrects clinical trial sponsor in ninth paragraph.) A failed prostate cancer drug might reverse Alzheimers. A former lung disease medicine may stop the ravages of muscular dystrophy. An experimental heartburn treatment may be better suited for treating a chronic cough. Those are the ideas that scientists have come up with for compounds sitting on AstraZeneca Plc (AZN)s shelf that the company made available to academics through a partnership with the U.K.s Medical Research Council. The council said today 15 research projects have been awarded 7 million pounds ($11.3 million) to study the drugs in animals or humans. The collaboration is part of an effort by AstraZeneca to take a second swing at products that failed in clinical trials and are outside the areas its focused on in the past few years, such as cancer and cardiovascular disease. While the money involved is small, AstraZeneca hopes to unearth something big. Theres a precedent: Viagra, which Pfizer Inc. (PFE) developed as a heart drug, was ultimately approved for erectile dysfunction and generated almost $2 billion in sales last year. We dont want to leave value on the table outside of the core business, Clive Morris, the AstraZeneca executive who heads up the … Continue reading →

MUSCULAR DYSTROPHY ASIANET NEWS From:kvs267Views:211 0ratingsTime:01:31More inPeople Blogs Excerpt from: MUSCULAR DYSTROPHY ASIANET NEWS - Video … Continue reading →