Category Archives: Muscular Dystrophy Treatment

The Muscular Dystrophy Association issued fun arrest warrants for about 100 local business and civic leaders April 26. Their crimes included having big hearts and being heavily armed with kindness. Their time "behind bars" was served at Stonewood Grill and Tavern, 10120 Forest Hill Blvd., during the MDA's Lock-Up Fundraiser. "We host the event every other year here in Wellington," said Brandy Miller, director of business development for the Greater Palm Beaches and Treasure Coast District MDA. "All the money that is raised stays local to help families pay for treatment and for scholarship to the MDA summer camp." The "jailbirds" helped raise more than $35,000 for families affected by neuromuscular diseases in the Wellington area. In order to be released, each jailbird's "bail" was set at $4,000 and the money was raised by encouraging friends, family, co-workers and business contacts to make contributions to MDA. The event was a success, and was filled with local leaders joining in on the fun. "All of the jailbirds had a great time and were very dedicated to raising much needed funds," Miller said in a press release. "Stonewood Grill and Tavern did an amazing job as our host site and it was … Continue reading →

By Brandon Santiago Hanford Sentinel | Posted: Saturday, April 28, 2012 7:45 am | HANFORD Local firefighters took to the streets Friday evening to battle not a fire, but a disease that affects millions every day muscular dystrophy as part of the annual Fill The Boot fundraiser. They were out walking the sidewalks and the medians asking pedestrians and drivers to donate. They carried signs and chanted Help out the MDA. Drivers honked their horns of support and rolled their windows down to give their spare change, or sometimes a 10- or 20-dollar bill. For the past eight years, the Hanford Fire Department has partnered with the Muscular Dystrophy Association (MDA) to help raise money for research and treatment of the disease. The concept of the fundraiser is very simple: Any one can give, whatever amount they want to the firefighters walking the streets holding out their boots. For the first time ever, Hanford Fire will be out collecting for two days. The campaign began Friday with department members on the corner of 12th Avenue and Mall Drive and fire academy students at the intersection of 11th Avenue and Lacey Boulevard. Today, the collection continues with firefighters only at the … Continue reading →

NEW YORK, April 19, 2012 /PRNewswire/ --N-Gene Research Laboratories, Inc. ("N-Gene"), today announces that Australian scientists in close cooperation with N-Gene have shown that BGP-15, a heat shock protein inducer, may be a novel therapy for treating the deadly, rare disease, Duchenne Muscular Dystrophy (DMD), as recently reported in the journal Nature. Currently there is no cure and no adequate therapy for DMD. BGP-15 was not only able to make a key protein functional again, thereby reducing muscle damage, but also increase the strength and endurance, and ultimately the lifespan, of DMD animals. "This pioneering work strengthens N-Gene's roleat the forefront of heat shock protein science," said Gabor K. Kalman, chief executive officer of N-Gene. Peter Literati, Ph.D., co-founder and chief scientific officer of N-Gene said, "We believe this publication reinforces the expectation that N-Gene's platform technology, based on stress-response regulation, will eventually result in the emergence of a novel drug class with diverse therapeutic directions. We are remaining focused on advancing BGP-15 for the treatment of type 2 diabetes, but we look forward to seeking partners with which to advance this technology and realize its potential in the treatment of DMD as well as many other disease indications." Dr. … Continue reading →

Public release date: 18-Apr-2012 [ | E-mail | Share ] Contact: Mike Wolterbeek mwolterbeek@unr.edu University of Nevada, Reno RENO, Nev. The nation's leading scientists and clinicians exploring treatment breakthroughs for congenital muscular dystrophy will convene April 22-24 on the campus of the University of Nevada, Reno, where research by Dean Burkin has led to a potential therapy. At the Myomatrix 2012 conference, experts will share their latest data and findings both published and soon to be published research seeking ways to build on recent progress. Burkin, a pharmacological researcher and faculty member at the University of Nevada School of Medicine, will present his work on laminin-111, a naturally occurring protein showing promise as a therapy for muscular dystrophy. Burkin's published research showed this protein is quickly picked up in the bloodstream of mice and prevents muscle damage. Initially shown to be an important finding for Duchenne muscular dystrophy, the most common form of muscular dystrophy, Burkin's continued research, recently published in the American Journal of Pathology, shows laminin-111 to also be an effective protein substitution therapy for congenital muscular dystrophy in mice. The University of Nevada, Reno's Tech Transfer Office has licensed the patent for laminin-111 to Prothelia, a Boston … Continue reading →

By Namitha Jagadeesh - Mon Apr 02 12:06:26 GMT 2012 U.S. stock-index futures declined as investors awaited a report that may show manufacturing expanded in the worlds biggest economy. Groupon Inc. sank 10 percent after the company said fourth- quarter sales were lower than previously stated. Avon Products Inc. (AVP) surged 24 percent after Coty Inc. offered to acquire the door-to-door cosmetics seller. Standard & Poors 500 Index (SPM2) futures expiring in June lost 0.1 percent to 1,401.70 at 8:04 a.m. in New York. The S&P 500 completed its biggest first-quarter rally since 1998 on March 30. Dow Jones Industrial Average futures slipped 18 points, or 0.1 percent, to 13,124 today. Theres a bit of concern that the U.S. economic recovery is starting to even out, and thats reflected in the markets, said Michael Hewson, a market analyst at CMC Markets in London. It really depends on what we get from the manufacturing survey today. The Institute for Supply Managements factory index -- scheduled for release at 10 a.m. New York time -- climbed to 53 in March from 52.4 in February, according to the average economist forecast in a Bloomberg News survey. Readings above 50 signal growth. A Commerce … Continue reading →

SOURCE: AVI BioPharma, Inc. BOTHELL, WA--(Marketwire - Apr 2, 2012) - AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based therapeutics, today announced that treatment with eteplirsen met the primary efficacy endpoint in a randomized, double-blind, placebo-controlled Phase IIb study in boys with Duchenne muscular dystrophy (DMD). Eteplirsen administered once weekly at 30mg/kg over 24 weeks resulted in a statistically significant (p 0.002) increase in novel dystrophin (22.5% dystrophin-positive fibers as a percentage of normal) compared to no increase in the placebo group. "This study represents a major advance in the field of DMD research as the results indicate that eteplirsen is producing consistent levels of dystrophin, which is the essential protein that these patients need," said Jerry Mendell, M.D., Director of the Centers for Gene Therapy and Muscular Dystrophy at Nationwide Children's Hospital and principal investigator of the Phase IIb study. Dr. Mendell added, "We anticipate that these levels of dystrophin could lead to significant clinical benefit if maintained over a longer course of treatment." In the study, a shorter duration of eteplirsen treatment, 12 weeks, did not show a significant increase in novel dystrophin (0.79% dystrophin-positive fibers as a percentage of normal; p-value NS), despite administration of the … Continue reading →

NEW YORK (Reuters) - AVI BioPharma Inc's shares could get a boost if its drug targeting treatment for a rare fatal form of muscular dystrophy proves to be effective in clinical drug trials, according to an article in the April 2 edition of Barron's. Clinical trial results for AVI's drug Eteplirsen were expected later this month, Barron's said. The drug is designed to treat Duchenne muscular dystrophy, a disease that develops in early childhood and most often results in the death by age 30. The disease strikes only boys, about one in 3,500, and affects more than 30,000 people in the United States, Europe and Japan. Favorable results could boost AVI's shares, which have doubled this year, Barron's said. Its shares closed at $1.54 on the Nasdaq on Friday. ThinkEquity analyst Marko Kozul has targeted AVI shares at $4, Barron's said. AVI also was developing drugs for Ebola and Marburg viruses with U.S. Department of Defense funding, Barron's said. (Reporting By Ilaina Jonas; Editing by Maureen Bavdek) Read the original here: Drug for rare disease may lift AVI BioPharma shares: Barron's … Continue reading →

SOURCE: AVI BioPharma, Inc. BOTHELL, WA--(Marketwire - Mar 30, 2012) - AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based therapeutics, today announced it will hold a conference call at 8:00 a.m. EDT (5:00 a.m. PDT) on Monday, April 2, 2012 to discuss top-line results from its Phase IIb study evaluating eteplirsen for the treatment of Duchenne Muscular Dystrophy (DMD). The conference call may be accessed by dialing 800.561.2718 for domestic callers and 617.614.3525 for international callers. The passcode for the call is 99858553. Please specify to the operator that you would like to join the "AVI BioPharma Phase IIb Top-Line Data Results Call." The conference call will be webcast live under the events section of AVI's website at http://www.avibio.com and will be archived there following the call for 90 days. Please connect to AVI's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary. About AVI BioPharma AVI BioPharma is focused on the discovery and development of novel RNA-based therapeutics for rare and infectious diseases, as well as other select disease targets. Applying pioneering technologies developed and optimized by AVI, the Company is able to target a … Continue reading →

Leading Duchenne-Specific Nonprofit Urges Congress to Enact as Part of User Fee Bill HACKENSACK, N.J., March 29, 2012 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) the leading advocacy organization fighting to end Duchenne muscular dystrophy has endorsed legislation that would shorten the amount of time the Food and Drug Administration (FDA) takes to review candidate therapies for life-threatening conditions like Duchenne that lack other treatment options. (Logo: http://photos.prnewswire.com/prnh/20100119/DC39975LOGO) The bill, known as the Faster Access to Specialized Treatments or FAST Act, is bipartisan legislation sponsored by Rep. Cliff Stearns of Florida and Rep. Edolphus Towns of New York. "For 20 years, FDA has had tools at its disposal to accelerate the agency's review of therapies targeted to treat life-threatening conditions that lack any other therapeutic options. Unfortunately, these tools have not yielded optimal advances despite the significant need that exists for patients and families living with Duchenne and other conditions," Pat Furlong, Founding President and CEO of PPMD, said. "The FAST Act is commonsense legislation that would leverage the Fast Track and Accelerated Review options at FDA's disposal. It would allow sponsors of candidate drugs to petition FDA to designate their product as being eligible for Fast Track review. It … Continue reading →

Public release date: 27-Mar-2012 [ | E-mail | Share ] Contact: Karim Wahbi, M.D. karim.wahbi@cch.aphp.fr JAMA and Archives Journals Patients with a cardiac irregularity and myotonic dystrophy type 1 (a severe neuromuscular disorder with a high risk of sudden death) who received an invasive treatment strategy that included testing of their heart's electrical conduction system and if needed, implantation of a device such as a pacemaker, had an associated higher rate of 9-year survival compared to patients treated noninvasively, according to a study in the March 28 issue of JAMA. Myotonic dystrophy type 1 (DM 1) is the most common inherited neuromuscular disease in adults, with an incidence of 1 in 8,000. "The manifestations of the disease include muscle weakness, myotonia [abnormally long muscular contractions], multiple endocrine disorders, respiratory insufficiency, and cardiac abnormalities. The prevention of sudden death is central to patient management," according to background information in the article. Progression of conduction system (special muscle fibers that conduct electrical impulses throughout the muscle of the heart) disease to complete atrioventricular block (impairment of the conduction between the atria and ventricles of the heart) is the presumed cause of sudden death in a high proportion of patients. Permanent cardiac pacing … Continue reading →