July 16, 2014
Researchers flex new muscle in SMA drug development
By Paige Blankenbuehler Lauren and Claire Gibbs share contagious laughter, ambition and a charismatic sarcasm. Both are honor students at Shawnee Mission East High School in a Kansas City suburb. They also share a neuromuscular disease called spinal muscular atrophy (SMA), designated as an “orphan disease” because it affects fewer than 200,000 people in the U.S. However, the landscape for individuals with SMA is quickly changing with the development of new drugs. More than 7 million people in the United States are carriers (approximately 1 in 40) of the so-called “rare” neurodegenerative disease, SMA. Lauren,17 (left) and Claire, 16 (right),…
