Donald S. Wood, PhD, President and CEO, MDA, emphasized the essential role of nonprofit organizations and their supporters in advancing treatments for rare diseases. "The approval of Soliris for pediatric myasthenia gravis is a major milestone—proof of how far science has come thanks to donors and supporters who generously give to organizations like Muscular Dystrophy Association because they believe in the power of scientific possibility. By funding research, advocating for the needs of the community, and working alongside our industry partners, MDA and our colleagues at organizations worldwide are pushing the boundaries to bring life-changing therapies to all people living with neuromuscular diseases."