Latest Biostrophin Stories
AMSTERDAM, October 13 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today that the European Medicines Agency has granted Orphan Drug Designation to AMT's gene therapy product AMT-080 for the treatment of Duchenne muscular dystrophy. Orphan Drug Designation for Duchenne muscular dystrophy (DMD) entitles AMT to ten year market exclusivity in Europe following marketing approval for AMT-080 if this...
Researchers at the University of Minnesota Medical School have discovered a new therapy that shows potential to treat people with Duchenne muscular dystrophy, a fatal disease and the most common form of muscular dystrophy in children.In the mouse model, researchers were able to substitute for the missing protein "“ dystrophin, which forms a key part of the framework that holds muscle tissue together "“ that results in the disease, effectively repairing weakened muscle tissue.Researchers...
Former New York Giants Jim Burt and Bart Oates Help Raise Money and Awareness for Duchenne Muscular Dystrophy FORT LEE, N.J., April 30 /PRNewswire-USNewswire/ -- Pat Furlong, Founding President and CEO of Parent Project Muscular Dystrophy (PPMD) announced today that former New York Giant stars Jim Burt and Bart Oates will join hundreds of guests for the 1st Annual Pour for a Cure, supporting PPMD, at the Hyatt Regency in New Brunswick, New Jersey on May 8, 2009. More than just a wine...
U.S. scientists say they've conducted the first gene therapy trial for a form of muscular dystrophy that disables by weakening hip and shoulder muscles. Researchers from the University of Florida, Nationwide Children's Hospital in Columbus, Ohio, and Ohio State University said they safely transferred a gene to produce a protein necessary for healthy muscle fiber growth into three teenagers with limb-girdle muscular dystrophy. We think this is an important milestone in establishing the...
Finding lays the foundation for human testingUsing a novel genetic technology that covers up genetic errors, researchers funded in part by the National Institutes of Health have developed a successful treatment for dogs with the canine version of Duchenne muscular dystrophy, a paralyzing, and ultimately fatal, muscle disease.The technology, known as "exon skipping" uses tailor-made snippets of DNA-like molecules as molecular "patches." These patches cover up mutant DNA...
Drugs similar to 1 in trials for heart disease may slow muscle loss in most common form of muscular dystrophyBased on a striking similarity between heart disease and Duchenne muscular dystrophy, researchers at Columbia University Medical Center have discovered that a new class of experimental drugs for heart failure may also help treat the fatal muscular disorder.At first glance, heart failure and the muscle-wasting Duchenne disease couldn't appear more dissimilar. Duchenne affects boys...
Brown University to Receive Grant Promoting Translational Research in Rare Muscular Disease MIDDLETOWN, Ohio, Dec. 23 /PRNewswire-USNewswire/ -- Patricia A. Furlong, Founding President and CEO 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 the first grant recipient of the newly launched End Duchenne Grant Award Program, Brown University. Brown...
Parent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne), will host its 14th annual CONNECT conference in Philadelphia, PA on July 17-20, 2008. The CONNECT conference brings together patients, scientists, physicians and advocates to share the latest research on Duchenne, the most common form of childhood muscular dystrophy. Duchenne is a progressive and fatal muscle disorder...
