Category Archives: Muscular Dystrophy Treatment

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Prosensa Holding NV (RNA) plunged 70 percent after the Dutch companys experimental muscular dystrophy drug failed to help patients walk better in a clinical trial. Sarepta Therapeutics Inc. (SRPT), the maker of another experimental medicine for the disease, soared. Prosensa dropped to $7.14 at the close in New York, with trading volume 87 times the three-month daily average. The Leiden, Netherlands-based companys shares are listed on the Nasdaq Stock Market. Sarepta, based in Bothell, Washington, jumped 18 percent to $43.30 for the biggest gain in more than 10 months. Prosensa is developing the treatment, drisapersen, with London-based GlaxoSmithKline Plc. (GSK) In a late-stage study dubbed Demand III, drisapersen failed to help patients more than a placebo in a test of six-minute walking distance, the companies said today in a statement. The results call into question the future of the drug, as the partners said they will conduct a full evaluation of the benefit-to-risk profile across all studies that will be completed by the end of 2013. While we are disappointed that this study did not meet its primary endpoint, we remain committed to the overall program and will continue to work closely with GSK, Prosensa Chief Executive Officer Hans Schikan … Continue reading →

Home Mail News Sports Finance Weather Games Groups Answers Screen Flickr More omg! Shine Movies Music TV Health Shopping Travel Autos Homes Mobile Yahoo News Search News Search Web Sign In Mail Help Account Info Help Suggestions Yahoo News Home Video Photos GMA Year in Review Odd Comics Travel Opinion Trending Now Who Knew? Weather The Upbeat U.S. U.S. Video GMA Education Religion Crimes and Trials Local Contributor Network Year In Review World World Video Middle East Europe Latin America Africa Asia Canada Australia/Antarctica Business Video Exclusives Today's Markets Stocks Personal Finance Marketplace Entertainment Video Clinton Concert Celebrity TV Movies Music Fashion Books Arts Theater Dear Abby Comics Odd News Sports Video NFL MLB NBA NCAAF NCAAB Soccer Cycling NHL Tennis Golf Boxing Motor Sports MMA Olympics Tech Gadgets Wireless Apple Social Media Security Open Source Gaming Apps This Could Be Big Upgrade Your Life Politics Remake America The Issues Women and Politics Press Releases Video Science Science Video Weather News Space / Astronomy Pets Dinosaurs / Fossils Biotech Energy Green Health Video Weight Loss Cancer Sexual Health Medications/Drugs Parenting/Kids Seniors/Aging Diseases/Conditions Blogs The Sideshow Katie's Take Power Players This Could Be Big Newsmakers Trending Now The Upbeat Who Knew? … Continue reading →

Sep. 17, 2013 A preclinical study led by researchers at Children's National Medical Center has found that a new oral drug shows early promise for the treatment of Duchenne muscular dystrophy (DMD). The results, published in EMBO Molecular Medicine, show that the drug, VBP15, decreases inflammation and protects and strengthens muscle without the harsh side effects linked to current treatments with glucocorticoids such as prednisone. Duchenne muscular dystrophy results in severe muscle degeneration and affects approximately 180,000 patients worldwide, mostly children. The current standard treatment uses glucocorticoids, but is limited due to serious side effects leading to fragile bones and suppression of both the immune system and growth. "These findings, while preliminary, are very promising for advancing the treatment of this disease, which causes disability in so many children worldwide," said Eric P. Hoffman, PhD, director of the Center for Genetic Medicine Research at Children's National. "The study also suggests the potential for new strategies in very early treatment, which could further benefit patients." The Children's National research team also observed that VBP15 inhibits the transcription factor NF-B, a key cell-signaling molecule found in most cell types that plays a role in inflammation and tissue damage. The team previously found … Continue reading →

Public release date: 17-Sep-2013 [ | E-mail | Share ] Contact: Emily Hartman ehartman@childrensnational.org 202-476-4500 Children's National Medical Center WASHINGTON, DC A preclinical study led by researchers at Children's National Medical Center has found that a new oral drug shows early promise for the treatment of Duchenne muscular dystrophy (DMD). The results, published in EMBO Molecular Medicine, show that the drug, VBP15, decreases inflammation and protects and strengthens muscle without the harsh side effects linked to current treatments with glucocorticoids such as prednisone. Duchenne muscular dystrophy results in severe muscle degeneration and affects approximately 180,000 patients worldwide, mostly children. The current standard treatment uses glucocorticoids, but is limited due to serious side effects leading to fragile bones and suppression of both the immune system and growth. "These findings, while preliminary, are very promising for advancing the treatment of this disease, which causes disability in so many children worldwide," said Eric P. Hoffman, PhD, director of the Center for Genetic Medicine Research at Children's National. "The study also suggests the potential for new strategies in very early treatment, which could further benefit patients." The Children's National research team also observed that VBP15 inhibits the transcription factor NF-B, a key cell-signaling molecule … Continue reading →

IRVINE, Calif. (PRWEB) September 16, 2013 The FSH Society announced that the Fourth Annual Celebrity Walk n Roll fundraiser, hosted by Max Adler of Glee, will take place on Sunday, October 6, 2013 at Heritage Park in Irvine from 10:00 a.m. to 2:00 p.m. More than 20 celebrities and athletes will participate in the walk and festivities to raise funds for FSH muscular dystrophy research. FSHD, one of the most prevalent types of muscular dystrophy, is a degenerative muscle disease that causes progressive weakness, usually starting with the face, shoulder and arms, but which can strike almost any skeletal muscle. FSHD affects approximately 500,000 people worldwide and between one and two percent of the population carries a genetic trait that places future generations at risk of the disease. Currently, there is no treatment. This annual event is not only a fundraiser for much needed research and advocacy, but a time for the community to come together and learn about a disease that affects the lives of many, said Amy Bekier, Celebrity Walk n Roll Chairwoman and Organizer. We are thrilled to have so many celebrity participants this year and we know this event will be a success. Celebrity guests include: … Continue reading →

Prosensa (NASDAQ: RNA) is a Dutch biotechnology company engaged in the discovery and development of RNA-modulating therapeutics for the treatment of genetic disorders. Our primary focus is on rare neuromuscular and neurodegenerative disorders with a large unmet medical need, including Duchenne muscular dystrophy, myotonic dystrophy and Huntingtons disease. We completed an IPO onto NASDAQ in June of this year. Prior to last years BIO Investor Forum in San Francisco, we were still in the very early stages of contemplating an IPO onto the U.S. markets. Our decision to list in the U.S. markets was not an easy one. Given a European biotech company had not accomplished its primary listing on NASDAQ in almost a decade, it was imperative that we gain exposure with U.S.-based investors prior to embarking on the road to an IPO. As much of the investment in the biotech industry is concentrated in the U.S., we felt that listing on NASDAQ would provide us with the necessary access to capital to be able to further develop our DMD portfolio of 6 unique compounds. BIOs legislative efforts leading to the passage of the JOBS Act in 2012 have been very supportive for IPOs. We evaluated several conferences that … Continue reading →

Public release date: 9-Sep-2013 [ | E-mail | Share ] Contact: Barry Whyte communications@embo.org 49-622-188-91108 European Molecular Biology Organization HEIDELBERG -- A preclinical study led by researchers in the United States has found that a new oral drug shows early promise for the treatment of muscular dystrophy. The results, which are published today in EMBO Molecular Medicine, show that VBP15 decreases inflammation in mice with symptoms similar to those found in patients with Duchenne muscular dystrophy. The authors found that the drug protects and strengthens muscle without the harsh side effects linked to current treatments with glucocorticoids such as prednisone. Duchenne muscular dystrophy results in severe muscle degeneration and affects approximately 180,000 patients worldwide, mostly children. Treatment with the current standard therapy, glucocorticoids, can only be used for a short time due to serious side effects leading to fragile bones and suppression of both the immune system and growth hormone production. The researchers also observed that VBP15 inhibits the transcription factor NF-kB, a key cell-signaling molecule found in most animal cell types that plays a role in inflammation and tissue damage. The study authors previously found out that NF-kB is active in dystrophin-deficient muscle years before the onset of symptoms, … Continue reading →

Parent Project Muscular Dystrophy logo. (PRNewsFoto/Parent Project Muscular Dystrophy) Pat Furlong to Speak at NINDS Nonprofit Forum & FDA Patient Network HACKENSACK, N.J., Sept. 9, 2013 /PRNewswire-USNewswire/ --Parent Project Muscular Dystrophy (PPMD) the leading advocacy organization fighting to end Duchenne muscular dystrophy and working to improve the quality of life and health outcomes of all patients with Duchenne has been invited to present at two upcoming stakeholder meetings. (Logo: http://photos.prnewswire.com/prnh/20100119/DC39975LOGO) The invitations, to PPMD Founding President & CEO Pat Furlong, were extended by leaders in the U.S. government in recognition of the organization's groundbreaking work to advance the patient and parent perspective in the drug development and review process. On September 10th, Furlong will speak at the Food and Drug Administration's (FDA's) Patient Network about the role patients and patient advocacy organizations must play to educate drug developers and regulators on a condition and its natural history, to offer perspectives on endpoints and trial design and to recruit clinical trial participants. The following day, Furlong will present at the plenary session of theNational Institute of Neurological Disorders and Stroke (NINDS)Nonprofit Forum, an annual meeting attended by dozens of leaders of patient advocacy and related organizations. Furlong will speak about PPMD's … Continue reading →

Leiden, The Netherlands, Sept. 6, 2013 (GLOBE NEWSWIRE) -- Prosensa Holding N.V. (RNA), the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today announced the dosing of the first patient in its Phase I/II clinical trial of PRO053, an exon-skipping compound for the treatment of Duchenne muscular dystrophy (DMD). "We are pleased that our fourth exon-skipping compound, PRO053, has advanced into clinical trials." Giles Campion, Prosensa's Chief Medical Officer, said. "Bringing new drug candidates, such as PRO053, into the clinic exemplifies how we can leverage our scientific and regulatory expertise gleaned from the experience in advancing the investigational development compound, drisapersen, through the clinic. Most importantly, a broader clinical portfolio may offer additional patients with subtypes of DMD a much-needed potential treatment option." PRO053, the company's fourth drug development candidate, induces exon 53 skipping and may be applicable to approximately 8% of all DMD patients. This is an open-label study designed to assess the safety, efficacy, tolerability and pharmacokinetics of multiple doses of PRO053 in a subset of patients with DMD. The primary outcome measure of the study will be change from baseline in the six-minute walk test (6MWT) after 48 weeks of treatment. … Continue reading →