Last week at the Goldman Sachs Healthcare Conference in Rancho Palos Verdes, Calif., Sarepta Therapeutics Inc. CEO Douglas Ingram noted that "more than a kid a day dies" from Duchenne muscular dystrophy (DMD) in the U.S., adding that officials at his Cambridge, Mass.-based firm "feel an enormous obligation to move heaven and earth to get this [gene] therapy to the community if it's possible" and quickly.