Political Quotes

“the fight against muscular dystrophy is a cause I have championed since my days in the House of Representatives. My fight against Duchenne muscular dystrophy began when a parent told me about his son's diagnosis with the disease. This parent refused to accept that there was no hope. The House and Senate agreed with the MD-CARE Act and, since that time, the life expectancy of the average Duchenne muscular dystrophy patient has increased by a full decade. This is progress we have made on behalf of sick people whose lives were threatened, and this is an example of government at its best. On Monday of this week, I saw the same devotion in the hundreds of Duchenne families who attended a meeting of the advisers of the Food and Drug Administration. The meeting's attendance broke records. I thank the FDA for making the appropriate accommodations to handle a crowd of this size. Some 11,000 people also tuned in remotely, watching the meeting via live stream. Monday's gathering was about what could be the first disease- modifying therapy for Duchenne muscular dystrophy. For more than 3 hours, the advisory committee heard from parents, doctors, and patients about the drug's impact on their lives. The stories were heartfelt and hopeful, reinforcing the importance of patient engagement in the drug approval process. The dedication of the Duchenne community continues to set an example for advocates of other rare diseases.…”