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One of the nation’s pre-eminent genetic researchers, Eric Hoffman, PhD, of Children’s Research Institute at Children’s National Medical Center, predicts that in relatively short order, medicine’s next innovation–individualized molecular therapies–will have the unprecedented ability to treat muscular dystrophies, and other disorders. In the latest edition of the New England Journal of Medicine, Dr. Hoffman posits [...]

A research team from Wake Forest University School of Medicine is the first to show that injections of a protein normally found in human cells can increase lifespan and delay the onset of symptoms in mice with ALS (amyotrophic lateral sclerosis), or Lou Gehrig’s disease. Reporting in the Nov. 28th issue of the Journal of [...]

Researchers at the University of Rochester Medical Center have identified a compound that eliminates myotonia — a symptom of muscular dystrophy — in mice. The study was published online in the Journal of Clinical Investigation. Rochester scientists were able to design a synthetic RNA-based molecule that, when injected into mice with myotonic dystrophy, restored a [...]