BioMarin’s Big Bet: FDA Weighs Approval Of Potential Blockbuster First Drug For Fatal Muscular Disorder
When Nicolas Gonzalez was 4 years old, his babysitter noticed that he had trouble climbing stairs. Then on Halloween when Nicolas and his mother, Michelle, went trick or treating in their Montgomery, Illinois, neighborhood he gave up partway through and sat down in the middle of the sidewalk. That’s when Michelle realized something was wrong.