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Latest Duchenne muscular dystrophy Stories

2013-08-02 12:37:49

The substance protects the body's supply of a cell signal for muscle regeneration A trace substance in caramelized sugar, when purified and given in appropriate doses, improves muscle regeneration in a mouse model of Duchenne muscular dystrophy. The findings are published today, Aug. 1, in the journal Skeletal Muscle. Morayma Reyes, professor of pathology and laboratory medicine, and Hannele Ruohola-Baker, professor of biochemistry and associate director of the Institute for Stem Cell...

2013-07-15 12:25:15

DART's Phase 1b/2a Study to Determine Safety and Tolerability of Lead Drug Candidate, Help Validate New Endpoint for Future DMD Studies CAMBRIDGE, Mass., July 15, 2013 /PRNewswire/ --DART Therapeutics Inc., an innovative, new-model biotechnology firm focused on developing therapies for Duchenne muscular dystrophy (DMD), has initiated a phase 1b/2a study of its lead drug candidate, HT-100 (delayed-release halofuginone). The phase 1b study (with a six-month 2a extension) in...

2013-07-08 12:13:59

Children with Duchenne muscular dystrophy often die as young adults from heart and breathing complications. However, scientists have been puzzled for decades by the fact that laboratory mice bearing the same genetic mutation responsible for the disease in humans display only mild symptoms and no cardiac involvement. Now, researchers at the Stanford University School of Medicine have developed a mouse model that accurately mimics the course of the disease in humans. The study is the first...

2013-06-26 20:21:03

Largest MDA golf tournament in the U.S. has a combined contribution of $3.7 million GAINESVILLE, Ga., June 26, 2013 /PRNewswire-iReach/ -- Mansfield Oil held the 27th Annual Mansfield Oil Golf Classic on June 11th and raised over $500,000 for the Muscular Dystrophy Association (MDA), a record for this event. The Annual Mansfield Oil Golf Classic is the largest MDA golf tournament in the U.S., and since its inception 27 years ago, the event has raised a collective $3.7 million for the...

2013-06-26 12:53:53

A drug developed to treat genetic diseases such as Duchenne muscular dystrophy and cystic fibrosis may need a radical rethink. In a new study published on 25 June in the open access journal PLOS Biology, researchers question the mechanistic basis of the drug called PTC124 (also known as Ataluren), casting doubt as to whether it has the molecular effects that are claimed for it. This may have implications for its effectiveness in treating genetic diseases. An estimated 10% of all human...

2013-06-24 16:27:33

HACKENSACK, N.J., June 24, 2013 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) will return to Baltimore for their Annual Connect Conference, the largest, most comprehensive annual, international conference focused entirely on Duchenne muscular dystrophy (Duchenne), June 27 - 30, 2013. Researchers, scientists, and Duchenne experts from around the world travel to the Connect Conference every year to share with families the latest in muscular dystrophy research and...

2013-06-21 12:24:18

HACKENSACK, N.J., June 21, 2013 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) - the nation's leading voice for patients and families impacted by Duchenne muscular dystrophy (Duchenne) - is urging the Secretary of Health and Human Services to update the nation's organ transplantation polices to ensure pediatric patients with rare diseases are not negatively impacted by outdated regulations. In the letter, PPMD urged HHS Secretary Kathleen Sebelius to initiate a...

2013-06-17 12:26:33

The annual telethon will air in prime-time, Labor Day weekend, Sunday, Sept. 1 TUCSON, Ariz., June 17, 2013 /PRNewswire-USNewswire/ -- The Muscular Dystrophy Association's nationally broadcast telethon, one of America's longest-standing and beloved fundraising and entertainment events, will return this Labor Day weekend with a new broadcast home: the ABC Television Network. (Logo: http://photos.prnewswire.com/prnh/20120424/DC93207LOGO) The network has signed on to air the two-hour...

Promise In Duchenne Muscular Dystrophy Shown With Genetic Editing
2013-06-05 08:48:09

Duke University Using a novel genetic 'editing' technique, Duke University biomedical engineers have been able to repair a defect responsible for one of the most common inherited disorders, Duchenne muscular dystrophy, in cell samples from Duchenne patients. Instead of the common gene therapy approach of adding new genetic material to "override" the faulty gene, the Duke scientists have developed a way to change the existing mutated gene responsible for the disorder into a normally...

2013-04-29 12:28:51

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...