Category Archives: Muscular Dystrophy Treatment

TUCSON, Ariz., April 30, 2013 /PRNewswire-USNewswire/ -- With the help of strong and dedicated retail partners and their caring customers, the Muscular Dystrophy Association's vital research and health care services programs will continue to benefit MDA families across the nation, thanks to the $23.3 million raised in its annual MDA Shamrocks fundraising program this season. (Logo: http://photos.prnewswire.com/prnh/20120424/DC93207LOGO) The campaign, which is centered on St. Patrick's Day, continues its record-breaking fundraising success, with total numbers still to be finalized. "Our Shamrocks program allows our retail partners and their generous customers to play a key role in elevating the Association's mission to offer help and hope to the families we serve in every community," said MDA President and CEO Steven M. Derks. "Every Shamrock purchased can have a life-changing effect by helping to fund the most promising research into lifesaving treatments and cures for muscle disease. We can't thank our partners and the American public enough for their continued support." Monies raised also provide MDA families with essential health care service programs, such as MDA clinics where individuals can be seen by a multidisciplinary team specializing in neuromuscular diseases. MDA clinics are at the forefront of research and treatment, and often serve … Continue reading →

HACKENSACK, N.J., April 29, 2013 /PRNewswire-USNewswire/ --Parent Project Muscular Dystrophy (PPMD), the leading nonprofit in the United States focused entirely on Duchenne muscular dystrophy (Duchenne), launched the Duchenne Central mobile app, in collaboration with Siren Interactive, a digital relationship marketing agency focused on rare disorder therapies. The app, which is available as a free download for Apple and Android devices, is an easy-to-use and on-the-go tool to locate clinical trials and clinics and view and share the latest Duchenne news. Duchenne is a fatal genetic disorder that slowly robs young men of their muscle strength. "Currently, there are no FDA-approved treatments for Duchenne; however, there are several potential therapies in development and currently in clinical trials," notes Pat Furlong, founding president and CEO of PPMD. "It's essential that patients and their families have ongoing access to information on the latest clinical trials. We're proud to bring this new resource to the Duchenne community." The app was developed with the goal of filling a need for the Duchenne community and developed with the experts at PPMD. Especially for rare disorders, enrolling enough patients in a clinical trial can be challenging. "With the explosive growth of mobile devices, people are using their … Continue reading →

67 WALL STREET, New York - April 29, 2013 - The Wall Street Transcript has just published its Biotechnology and Pharmaceuticals Report offering a timely review of the sector to serious investors and industry executives. This special feature contains expert industry commentary through in-depth interviews with public company CEOs and Equity Analysts. The full issue is available by calling (212) 952-7433 or via The Wall Street Transcript Online. Topics covered: Health Care - Biotechnology and Pharmaceuticals - Executive Officer Interviews - Biotechnology and Pharmaceutical Investing - Orphan Drug and Biologics Manufacturing - Biotechnology and Pharmaceutical Companies Valuation Companies include: Gilead Sciences Inc. (GILD), Idenix Pharmaceuticals Inc. (IDIX), Achillion Pharmaceuticals, Inc (ACHN), Vertex Pharmaceuticals Incorpo (VRTX), Cubist Pharmaceuticals Inc. (CBST), Optimer Pharmaceuticals, Inc. (OPTR), Theravance Inc. (THRX), InterMune Inc. (ITMN), GlaxoSmithKline plc (GSK), Eli Lilly & Co. (LLY), Merck & Co. Inc. (MRK), Alexion Pharmaceuticals, Inc. (ALXN) and many more. In the following excerpt from the Biotechnology and Pharmaceuticals Report, an expert analyst discusses the outlook for the sector for investors: TWST: Which kinds of treatments or specific drugs do you think represent the most promising opportunities that investors may want to take a closer look at? Mr. Skorney: One of … Continue reading →

Javascript is currently disabled in your web browser. For full site functionality, it is necessary to enable Javascript. In order to enable it, please see these instructions. 6 hours ago by Liz Ahlberg Illinois chemists -- from left, undergraduate Kali A. Miller, graduate students Amin Haghighat Jahromi and Lien Nguyen, graduate student in Chemistry and professor Steven C. Zimmerman developed a small-molecule compound that could lead to therapeutic treatment for myotonic dystrophy, an as-yet untreatable disease. Credit: Ben Woloszyn (Phys.org) There's hope for patients with myotonic dystrophy. A new small molecule developed by researchers at the University of Illinois has been shown to break up the protein-RNA clusters that cause the disease in living human cells, an important first step toward developing a pharmaceutical treatment for the as-yet untreatable disease. Steven C. Zimmerman, the Roger Adams Professor of Chemistry at the U. of I., led the group in developing and demonstrating the compound. The National Institutes of Health supported the work published in the journal ACS Chemical Biology. Myotonic dystrophy type 1 is the most common form of muscular dystrophy in adults, affecting one in 8,000 people in North America. It causes progressive weakness as the muscles deteriorate over time. … Continue reading →

HACKENSACK, N.J., April 23, 2013 /PRNewswire-USNewswire/ --Parent Project Muscular Dystrophy (PPMD) has released Putting Patients First , a white paper outlining recommendations to speed responsible access to new therapies for Duchenne muscular dystrophy (Duchenne) and other, rare, serious, and life-threatening neurological disorders. PPMD developed this white paper with an expert Advisory Committee on Policies to Promote Responsible Access to New Therapies, made up of leading authorities from the nonprofit, academic, medical, legal, and pharmaceutical sectors. Putting Patients First urges the U.S. Food and Drug Administration (FDA) to work with PPMD and the rare disease community to take advantage of opportunities created by the 2012 passage of the Food and Drug Administration Safety and Innovation Act to enhance patient access to new therapies for serious and life-threatening disorders. In implementing the law, PPMD and the Advisory Committee encourage the FDA to "strike a more appropriate balance between clinical certainty and patient access to potentially life-saving treatments." The white paper makes four recommendations: Pat Furlong, Founding President and CEO of PPMD, hopes that Putting Patients First will illustrate the need for the FDA to accelerate approval of potentially life-saving drugs intended to treat rare disorders, like Duchenne. Says Furlong, "We have been … Continue reading →

The Merced Fire Department didn't just "Fill The Boot" on Tuesday -- it overflowed the boot with more than $11,000 in donations, a record for the annual fund-raiser. The funds benefit the Muscular Dystrophy Association and help send local kids to an annual camp. More than 20 fire personnel volunteered to raise money for the association during the seven-hour event at G Street and Olive Avenue. "We've had a great turnout from our members which helps us collect more donations," said Fire Capt. Cory Haas. "Merced has traditionally done a great job supporting Muscular Dystrophy Association." The fund-raiser, a 59-year-tradition in cities across the country, is supported by the local firefighters union. Chad Englert, union president and Fire Department engineer, said the event would not be possible without the public's support. "We want to thank the community for donating. They're the ones that make it possible," he said. "We're just out here facilitating." Englert's daughter, 4-year-old Amanda, was a big helper during Tuesday's fund-raiser. "I want to help Dada," she said. As cars passed by on G Street, firefighter Jeff Trost, who helped organize the event, took the time to spread the word about the Muscular Dystrophy Association. He caught … Continue reading →

Apr. 24, 2013 Stanford University School of Medicine scientists have created a mouse model of muscular dystrophy in which degenerating muscle tissue gives off visible light. The observed luminescence occurs only in damaged muscle tissue and in direct proportion to cumulative damage sustained in that tissue, permitting precise monitoring of the disease's progress in the mice, the researchers say. While this technique cannot be used in humans, it paves the way to quicker, cheaper and more accurate assessment of the efficacy of therapeutic drugs. The new mouse strain is already being employed to test stem cell and gene therapy approaches for muscular dystrophies, as well as drug candidates now in clinical trials, said Thomas Rando, MD, PhD, professor of neurology and neurological sciences and director of Stanford's Glenn Laboratories for the Biology of Aging. Rando is the senior author of a study, published online April 24 in the Journal of Clinical Investigation, describing his lab's creation of the experimental mouse strain in which an inserted gene coding for luciferase, the protein that causes fireflies' tails to glow, is activated only in an important class of rare stem cells that, collectively, serve as a reserve army of potential new muscle tissue. … Continue reading →

For the 15th year in a row, Chris Stenmon rallies his friends and family in the fight against facioscapulohumeral muscular dystrophy (FSHD). This years event will be held on April 27th at Florian Hall in Dorchester. Boston, MA (PRWEB) April 22, 2013 "The surgeon took one look at me and left the room for five minutes and came back with a medical book with a picture of a teenager who looked very similar to me, with weak face, shoulder and upper arm muscles," recalls Stenmon. It turned out that he had facioscapulohumeral muscular dystrophy (FSHD). The diagnosis came as a shock to Stenmon and his family. But he vowed to not let his disease pin him to the mat. Next Saturday, April 27th, for the 15th year in a row, Stenmon is rallying hundreds of friends to his Friends Supporting Hope fundraiser. This years event takes place from 7 PM to midnight at Florian Hall (Boston Firefighters Local 718), 55 Hallet Street in Dorchester. Fox 25 News Commentator Doug VB Goudie is presiding as emcee. The Boston-based band Fenian Sons will rock the hall with its popular Irish sounds. In the 25 years since learning he had FSHD, Stenmon graduated … Continue reading →

SAN DIEGO, April 22, 2013 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc., a biopharmaceutical company engaged in the discovery and development of adult stem cell modulators, announced today the presentation of preclinical data from its WNT7a protein analog program for the treatment of muscular dystrophy at the Muscular Dystrophy Association (MDA) 2013 Scientific Conference, April 21-24, in Washington DC. The presentations describe the engineering of pharmaceutically optimized WNT7a protein analogs, as well as their mechanism of action and efficacy profile in preclinical pharmacology studies. "The data presented today provide strong preclinical support for the therapeutic potential of WNT7a analogs in muscular dystrophy, a complex group of disorders with a large unmet need for novel, differentiated and potentially complementary treatment approaches," commented Christian Weyer, M.D., M.A.S., President and Chief Executive Officer of Fate Therapeutics. "We are working towards the nomination of an investigational new drug (IND) candidate later this year, and are excited about the potential to advance this new mechanism toward clinical studies." In the MDX mouse model of muscular dystrophy, intramuscular injection of a novel WNT7a analog resulted in significant dose dependent muscle hypertrophy and several-fold expansion of the satellite stem cell population. Moreover, three weeks after a single intramuscular … Continue reading →

Sarepta Therapeutics Inc.'s study of its experimental Duchenne muscular dystrophy drug eteplirsen may be too small for the faster approval the company seeks from regulators, according to an analyst who lowered her rating on the stock. Shares slide in premarket trading Tuesday. The Cambridge, Mass., company said Monday after markets closed that the U.S. Food and Drug Administration asked for more information from existing data on eteplirsen to help with its decision on whether to approve the treatment. Janney Capital Markets analyst Kimberly Lee said in a Tuesday morning research note she could not rule out the possibility that Sarepta's study of 12 patients treated with eteplirsen is too small for accelerated approval. Investors didn't seem optimistic, either. Sarepta shares slid 6.7 percent, or $2.64, to $36.60 before markets opened Tuesday. A Sarepta spokesman did not immediately return a call from The Associated Press seeking comment. The FDA grants accelerated approval to promising medical products designed to treat serious diseases. The program allows the agency to clear innovative drugs based on promising preliminary results. Duchenne muscular dystrophy is a fatal, genetic disease that causes increasing muscle weakness and affects one of every 3,500 boys worldwide. Sarepta said death usually occurs … Continue reading →