Category Archives: Muscular Dystrophy Treatment

01-10-2012 06:54 Introduction of Ayurvedic Panchakarma by Dr Ravi Prasad (Head Of the Department ,PG Panchakarma, BRKR Ayurvedic College, Hyderabad) See the article here: Ayurvedic Panchakarma - Introduction - Treatment for Muscular Dystrophy - Video … Continue reading →

By Misti Crane The Columbus Dispatch Friday October 5, 2012 9:01 AM Parents of boys with Duchenne muscular dystrophy now have even more reason for hope. The company that makes an experimental drug designed to treat about 13percent of the approximately 12,000 U.S. boys with the disease has released new results from a small but important study that shows significant benefit from the drug eteplirsen. Dr. Jerry Mendell, director of the Center for Gene Therapy at Nationwide Childrens Hospitals research institute, is the lead researcher on the study of 12 boys with the debilitating, and ultimately deadly, disease. He and the company, Sarepta Therapeutics, previously shared optimistic news after observing the boys health about nine months into treatment with a weekly infusion of the drug. Now, they say they have seen significant benefit after almost a year. Sarepta Therapeutics said this week that the drug increases dystrophin, a vital protein missing in boys with the genetic disorder. It also slowed progression of the disease, as measured by how far the boys could walk in six minutes. The four boys who had the strongest dose for 48 weeks were able to walk an average of about 23 yards farther than when … Continue reading →

An experimental drug appears to preserve and possibly even improve the ability of boys with Duchenne muscular dystrophy to walk, according to the results of a clinical trial announced Wednesday, raising hopes that the first effective treatment for the disease might be on the horizon. Boys with the disease who received the highest dose of the drug had a slightly improved ability to walk after 48 weeks of treatment, the drug's developer, Sarepta Therapeutics, announced. By contrast, the boys who received a placebo suffered a sharp decline in how well they could walk. The drug, called eteplirsen, also appeared to restore levels of the key protein that muscular-dystrophy patients lack to about half of normal levels, Sarepta said. There are many caveats. The trial had only 12 patients, with four patients receiving the top dose and four the placebo, and the data has not been published or reviewed by experts. It is also unclear how long the effects of the drug will last or whether safety issues would arise with longer treatment. Even if it does work, the drug would be appropriate for only about 15 percent of patients with the disease, those with the particular genetic mutation the drug … Continue reading →

Sarepta Therapeutics Inc. (SRPT) tripled in trading after its drug for Duchenne muscular dystrophy helped patients walk farther in a study and restored a key protein lacking in some of those with the disease. Sarepta surged 200 percent to $44.93 at the close in New York, its biggest increase since 1997, after the Cambridge, Massachusetts-based biotechnology company said its experimental drug eteplirsen met the primary goal of the mid-stage study. Patients who took 50 milligrams of eteplirsen once a week for 48 weeks could walk 89 meters (292 feet) farther during a six-minute test than those who took a placebo for 24 weeks and the drug for 24 weeks, Sarepta said today in a statement. The medicine may offer new promise against a disease in which current therapy only extends life and make it easier, rather than reverses the effects. With this study the boys actually got stronger, a pretty impressive result, said Valerie Cwik, interim president of the Muscular Dystrophy Association in Tucson, Arizona. Eteplirsen repairs a gene mutation in about 13 percent of Duchenne muscular dystrophy patients, helping them produce a missing protein and regain strength, Sarepta said in its statement. Current therapy includes prednisone, a corticosteroid that … Continue reading →

NEW YORK (AP) -- Shares of Sarepta Therapeutics Inc. nearly tripled in value Wednesday after the company reported positive clinical data for its Duchenne muscular dystrophy drug eteplirsen. Sarepta said eteplirsen slowed the progress of the disease and increased patients' levels of the protein dystrophin in an extension of a midstage trial. Low levels of that protein are the cause of Duchenne muscular dystrophy. Eteplirsen is Sarepta's most advanced drug candidate, and in morning trading the stock jumped $25.70 to $40.69. Shares of the Cambridge, Mass., company closed at $14.99 on Tuesday. On Wednesday the shares peaked at $43.10, their highest price since 2006. Duchenne muscular dystrophy is a condition that occurs in about 1 of every 3,600 male infants. The National Institutes of Health say patients typically die before the age of 25, and Sarepta tested the drug on 12 patients between the ages of 7 and 13. Shares of Sarepta had already more than doubled in value since July, when the company reported earlier data from the study. Sarepta also announced that the U.S. Department of Defense canceled a contract for the development of a treatment for Ebola virus. Work on the program was stopped in August because … Continue reading →

NEWPORT BEACH, Calif.--(BUSINESS WIRE)-- CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, is pleased by todays announcement by Sarepta Therapeutics (formerly AVI BioPharma) that its exon-skipping compound, eteplirsen, achieved significant clinical benefit after 48 weeks of treatment in a Phase IIb study in Duchenne muscular dystrophy. CureDuchenne, along with Children National Medical Center in Washington, D.C., and the Foundation to Eradicate Duchenne, provided funding in 2010 for this research to progress into human clinical trials. The eteplirsen drug helped increase the production of dystrophin, which is lacking due to a defect on the dystrophin gene that causes Duchenne, and slowed the progression of the disease, as measured by the 6-minute walk test. This is a milestone for the company and patients alike. The result will pave the way for an end of phase II meeting between the company and the FDA to discuss and agree to the plan for a phase III registration trial. Sarepta will scale up the manufacturing to accommodate a larger trial. This is an important day for Duchenne, said Chris Garabedian, CEO of Sarepta Therapeutics. We are grateful to CureDuchenne, and other organizations, for their support and … Continue reading →

By Brenton Flynn | More Articles October 3, 2012 | 87.4 meters. For Duchenne muscular dystrophy patients taking a 50 milligram dose of Sarepta Therapeutics' (Nasdaq: SRPT) eteplirsen for 48 weeks, that's how much longer the average patient walked during a six-minute walk test than a placebo group, which began taking the drug after 24 weeks. Not only did the 50mg group outperform placebo, but it showed an average 27.1 meter increase in distance walked over the treatment period -- an astonishing improvement over the 36-week data, which showed a 5.2 meter decline for the 50mg group. Not only did the walk test show a statistically significant benefit, but measures of dystrophin, a vital protein that protects muscle fibers, showed statistically significant increases in both the initial treatment and delayed treatment/placebo group. This is the kind of data that could merit an accelerated approval from the Food and Drug Administration. We'll have to wait to find out for sure, but shareholders are already piling in, bidding up shares by more than 110% in early morning trading. Foolish bottom lineOne thing's for sure: These results are revolutionary for the patients and families dealing with Duchenne. However, today's move could place the … Continue reading →

Rare Disease Industry Leaders and Advocates Honored; Recording Artists Chris Mann, Katrina Parker and Elliott Yamin Gave Touching Performances DANA POINT, Calif., Oct.2, 2012 /PRNewswire-USNewswire/ --On September 27, 2012, rare disease patients, advocates, patient organizations, government agencies, pharmaceutical companies, medical researchers, celebrities, Olympic champions and private sector representatives attended the Global Genes | R.A.R.E. Project (www.globalgenes.org) 1st Annual "RARE Tribute to Champions of Hope" benefit at the Balboa Bay Club & Resort in Newport Beach, CA. The evening was a star-studded affair to raise funds and awareness for rare and genetic diseases that impact 1 in 10 Americans and 350 million people globally. More than 400 guests attended the benefit and a number of awards were presented to celebrate the pioneering achievements of individuals and organizations championing the rare and genetic disease cause. "It was such an honor to celebrate the accomplishments of these remarkable rare disease award recipients," Nicole Boice, president of the Global Genes Project. "We are also incredibly grateful to the artists and stars who volunteered their time and talent to make our first event a tremendous success. Their support means the world to the millions of rare disease patients who know they are not alone in … Continue reading →

SOURCE: Sarepta Therapeutics, Inc. CAMBRIDGE, MA--(Marketwire - Oct 2, 2012) - Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, announced today it will hold a conference call at 8:00 a.m. EDT (5:00 a.m. PDT) on Wednesday, October 3, 2012 to discuss 48-week results from its Phase IIb study evaluating eteplirsen for the treatment of Duchenne muscular dystrophy (DMD). The conference call may be accessed by dialing 866.356.3093 for domestic callers and 617.597.5381 for international callers. The passcode for the call is 93880948. Please specify to the operator that you would like to join the "Sarepta Therapeutics 48-Week Results Call."The conference call will be webcast live under the events section of Sarepta's website at http://www.sareptatherapeutics.com and will be archived there following the call for 90 days. Please connect to Sarepta's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary. About Sarepta Therapeutics Sarepta Therapeutics is focused on developing first-in-class RNA-based therapeutics to improve and save the lives of people affected by serious and life-threatening rare and infectious diseases. The Company's diverse pipeline includes its lead program eteplirsen, for Duchenne muscular dystrophy, as well as potential … Continue reading →

29-09-2012 10:30 Dr. Nandini Gokulchandran talking about stem cell treatment for muscular dystrophy and showing video improvements of some patients who had undergone stem cell treatment. This interview was telecast ed on Zee 24 Tas. Stem Cell Treatment done at Neurogen ,Brain & Spine Institute, Surana Sethia Hospital and Research Centre, Suman Nagar, Sion-Trombay Road, Chembur,Mumbai-71. Tel: - +91 9920 200400, 022-25283706 Read more from the original source: MUSCULAR DYSTROPHY DAY SPECIAL INTERVIEW ON STEM CELL TREATMENT 9th September 2012. - Video … Continue reading →