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Last updated on April 16, 2014 at 1:21 EDT

Latest Duchenne muscular dystrophy Stories

2013-02-11 08:25:55

CAMBRIDGE, Mass., Feb. 11, 2013 /PRNewswire/ -- Catabasis Pharmaceuticals, Inc., today announced that a three-part series of Phase 1 studies showed that CAT-1004, a SMART Linker conjugate of docosahexaenoic acid (DHA) and salicylate created using the company's proprietary technology, was safe and well tolerated. Data also show that the conjugated compound was absorbed following oral administration and metabolized to its active components intracellularly, providing proof of concept for the...

Experimental Gene Therapy Treatment Offers Hope For Youngster With Duchenne Muscular Dystrophy
2013-02-09 09:05:38

UC Davis Health System [ Watch The Video ] Jacob Rutt is a bright 11-year-old who likes to draw detailed maps in his spare time. But the budding geographer has a hard time with physical skills most children take for granted “• running and climbing trees are beyond him, and even walking can be difficult. He was diagnosed with a form of muscular dystrophy known as Duchenne when he was two years old. The disease affects about 1 in 3,500 newborns “• mostly boys...

2013-02-07 12:26:14

Swimmer Travels to Capitol Hill to Urge Congress to Continue Support of People with Duchenne Muscular Dystrophy WASHINGTON, Feb. 7, 2013 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD), the leading non-profit in the United States focused entirely on Duchenne muscular dystrophy (Duchenne), welcomes 11-time Olympic medalist Ryan Lochte to their Annual Advocacy Conference in Washington, D.C., February 10-12, 2013. Lochte, one of the top American performers at the 2012...

2013-02-01 16:26:11

University of Florida to Receive $59,000 Grant from Duchenne-specific Organization HACKENSACK, N.J., Feb. 1, 2013 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) announced that it has funded $59,000 to Krista Vandenborne, PhD of the University of Florida in Gainesville to test the ability of magnetic resonance imaging (MRI) to detect changes in the muscles of those living with Duchenne muscular dystrophy (Duchenne) who participated in the Sarepta phase II trial of...

2013-01-18 08:23:43

Dr. Craig McDonald of UC Davis to Receive Supplemental Funds HACKENSACK, N.J., Jan. 18, 2013 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) has awarded Dr. Craig McDonald of the University of California, Davis (UC Davis) $175,000 in supplemental funds to expand his ongoing study through the 20 CINRG (Cooperative International Neuromuscular Research Group) centers to better understand the progression of Duchenne muscular dystrophy (Duchenne) and determine the impact...

2012-12-12 12:25:31

CLEVELAND, Dec. 12, 2012 /PRNewswire/ -- Milo Biotechnology today announced its AAV1-FS344 has been granted Orphan Drug designation from the FDA's Office of Orphan Products Development for treatment of Becker and Duchenne muscular dystrophy. AAV1-FS344 is a gene therapy-delivered myostatin inhibitor that increases muscle strength. The program is currently in a Phase I/II trial at Nationwide Children's Hospital in adult patients with Becker muscular dystrophy and inclusion body myositis, a...

2012-12-10 12:41:23

Approximately 250,000 people in the United States suffer from muscular dystrophy, which occurs when damaged muscle tissue is replaced with fibrous, bony or fatty tissue and loses function. Three years ago, University of Missouri scientists found a molecular compound that is vital to curing the disease, but they didn't know how to make the compound bind to the muscle cells. In a new study, published in the Proceedings of the National Academies of Science, MU School of Medicine scientists Yi...

2012-12-06 08:27:06

SOUTH PLAINFIELD, N.J., Dec. 6, 2012 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) today announced that the European Medicines Agency (EMA) has validated a Marketing Authorization Application (MAA) seeking conditional approval for ataluren, an investigational new drug for the treatment of patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). Validation of the MAA confirms that the submission is complete and begins the EMA's Committee for Human Medicinal Products' (CHMP) review...

2012-12-03 08:26:39

Furlong Will Address Role of Advocacy in Facilitating Basic Scientific Research HACKENSACK, N.J., Dec. 3, 2012 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) founding president and CEO Pat Furlong, has been invited to speak today at the Institute of Medicine's (IOM) Roundtable on Translating Genomic-Based Research for Health. IOM is holding a public workshop today in Irvine, CA titled "Improving the Efficiency and Effectiveness of Genomic Science Translation."...

2012-10-10 07:28:36

CAMBRIDGE, Mass., Oct. 10, 2012 /PRNewswire/ -- DART Therapeutics, LLC, an innovative, new-model biotechnology firm focused on developing therapies for Duchenne Muscular Dystrophy (DMD), announced today the successful completion of a study to evaluate a promising new biomarker for DMD. The biomarker, electrical impedance myography (EIM), is a non-invasive and simple-to-use technique that allows the operator to measure the health of a muscle and track its changes over time. The...