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BioMarin Pharmaceutical Inc. announced today that it has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for BMN-701, a novel fusion of insulin-like growth factor 2 and alpha glucosidase (IGF2-GAA) in development for the treatment of Pompe disease. An investigational new drug application (IND) for BMN-701 has been submitted, investigational material has been manufactured and a Phase I/II study is expected to start in the first quarter of 2011.
Moffitt Cancer Center, Sanford-Burnham Medical Research Institute and Florida Hospital have announced they will collaborate on the creation of a Personalized Medicine Partnership of Florida (PMP Florida). The partnership will conduct collaborative research to accelerate discovery and develop new treatments in the areas of cancer and metabolic diseases, including obesity, diabetes and cardiovascular disease.
New research from the University of Toronto could provide some restful nights for the 18 million North Americans who suffer from obstructive sleep apnea.
TIME magazine has named Penn Nursing's pioneering research on autism and low birthweight one of the "Top 10 New Findings in Parenting" of 2011. In October, Penn Nursing Professor Jennifer Pinto-Martin, PhD, MPH, and colleagues reported in Pediatrics that premature infants are five times more likely to have autism than children born at normal weight.
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