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Last updated on April 17, 2014 at 12:30 EDT

Latest Becker's muscular dystrophy Stories

2012-01-04 07:00:00

NEWTON, Mass., Jan. 4, 2012 /PRNewswire/ -- Halo Therapeutics, LLC, a clinical-stage biopharmaceutical company developing novel therapeutics for rare fibrotic diseases, today announced that the United States Food and Drug Administration (FDA) granted orphan drug designation for HT-100 for treatment of Duchenne muscular dystrophy (DMD). DMD is a progressive and fatal neuromuscular disorder, which afflicts approximately 1 in 3,500 boys worldwide. HT-100, also known as halofuginone, is an orally...

2011-12-19 11:22:00

TUCSON, Ariz., Dec. 19, 2011 /PRNewswire-USNewswire/ -- The Muscular Dystrophy Association (MDA) has awarded $750,000 to Summit PLC, a UK biotechnology company, to continue development of an experimental drug that may become a viable treatment for boys with Duchenne muscular dystrophy (DMD). The funding comes from MDA Venture Philanthropy (MVP), a part of MDA's translational research program. DMD is one of nine types of muscular dystrophy, a group of genetic, degenerative diseases primarily...

2011-12-14 02:22:16

People who have the most common type of adult muscular dystrophy also have a higher risk of getting cancer, according to a paper published today in the Journal of the American Medical Association. The team found that patients who have myotonic muscular dystrophy are at increased risk primarily for four types of cancer: brain, ovary, colon, and the uterine lining known as the endometrium. The team also found a possible increased risk for some other types of cancer, including cancer of the...

2011-12-01 10:52:46

Researchers at the University of North Carolina at Chapel Hill have shown that it is safe to cut and paste together different viruses in an effort to create the ultimate vehicle for gene therapy. In a phase I clinical trial, the investigators found no side effects from using a "chimeric" virus to deliver replacement genes for an essential muscle protein in patients with muscular dystrophy. "This trial demonstrates that gene therapy is no longer limited by the viruses we find in nature, and...

2011-11-21 09:50:59

An international team of researchers from Leeds, London and Berlin has discovered more about the function of muscle stem cells, thanks to next-generation DNA sequencing techniques. The work, which was co-led from the University of Leeds' School of Medicine and the Charité, Berlin, is published this week in the journal Nature Genetics. The researchers investigated several families whose children suffered from a progressive muscle disease. The children developed severe...

2011-10-27 11:30:00

Naperville's Karlin Family Continues Fight to End Duchenne on Behalf of Son NAPERVILLE, Ill. Oct. 27, 2011 /PRNewswire-USNewswire/ --Marty Karlin, a board member of Parent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne), announced today that his foundation, Rally for Ryan, will host its third annual Gala Event & Raffle at the White Eagle Golf Club in Naperville, Illinois on...

2011-10-13 13:10:00

Grant to Help Largest National Duchenne Organization Fund Research Projects HACKENSACK, N.J., Oct. 13, 2011 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne), announced that it has received a grant from the National Institutes of Neurological Disorders and Stroke (NINDS), part of the National Institutes for Health. This grant was awarded to fund...

2011-09-22 13:19:00

Largest National Duchenne Organization Gives $98,000 for Bridge Funding HACKENSACK, N.J., Sept. 22, 2011 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne), announced that it has awarded Tejvir S. Khurana, MD, PhD, Professor of Physiology at the University of Pennsylvania and Pennsylvania Muscle Institute an End Duchenne Grant for $98,000. (Logo:...

2011-09-07 11:32:00

Largest National Duchenne Organization Responds to FDA's "PDUFA Reauthorization Performance Goals and Procedures, Fiscal Years 2013 Through 2017" HACKENSACK, N.J., Sept. 7, 2011 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne), applauded the FDA's "PDUFA Reauthorization Performance Goals and Procedures, Fiscal Years 2013 Through 2017" (PDUFA V)....

2011-09-02 07:00:00

SOUTH PLAINFIELD, N.J. and CAMBRIDGE, Mass., Sept. 2, 2011 /PRNewswire/ -- PTC Therapeutics, Inc. and Genzyme, a Sanofi company, announced today the restructuring of their collaboration. Under the original agreement, PTC held commercial rights for the U.S. and Canada and Genzyme held commercial rights in all other countries. Under the restructured agreement, PTC regains worldwide rights to ataluren and Genzyme retains an option to commercialize ataluren in indications other than...