Amy Klobuchar: “I rise today as the cochair of the Rare Disease Congressional Caucus in recognition of patients with Duchenne muscular…”

“I rise today as the cochair of the Rare Disease Congressional Caucus in recognition of patients with Duchenne muscular dystrophy and the loved ones who care for them. Duchenne is a devastating, rare disease that primarily affects boys and young men. There is no cure. It is 100 percent fatal. There are no approved disease-modifying treatments at this time, but we want to give them hope. In 1999, there were no human clinical trials for Duchenne. Today, there are 22 observational trials currently underway. Life expectancy rates have increased by about 10 years in just the past decade. The FDA has more tools in its toolbox than ever to accelerate approvals of safe and effective Duchenne therapies, but we would like more therapies to be approved in the future. Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3,500 male children. The disease results in the gradual loss of muscle strength, usually beginning before age 5. The progressive muscle weakness leads to serious medical problems, particularly issues related to the hearts and lungs. By age 14, over 80 percent of these boys are using wheelchairs. My work on Duchenne muscular dystrophy began when I was elected to the Senate. It was an issue my dear friend and former Minnesota Senator, Paul Wellstone, championed.…”