[Congressional Record (Bound Edition), Volume 162 (2016), Part 4]
[House]
[Pages 5616-5617]
[From the U.S. Government Publishing Office, www.gpo.gov]




          GIVING HOPE TO DUCHENNE MUSCULAR DYSTROPHY PATIENTS

  (Mr. PAULSEN asked and was given permission to address the House for 
1 minute and to revise and extend his remarks.)
  Mr. PAULSEN. Mr. Speaker, I rise today to speak about a genetic 
disorder, Duchenne muscular dystrophy,

[[Page 5617]]

that has caused heartbreak for families across the country. The genetic 
mutation, found typically in boys, weakens muscles and leaves most 
sufferers confined to wheelchairs. Due to lung or heart failure, most 
afflicted with Duchenne pass away before their 25th birthday.
  However, there is hope with new medical advancement that could 
increase the lifespan and improve the quality of life for those living 
with Duchenne. A new drug was used in a medical trial where every 
participant was able to walk for 4 years after starting the treatment, 
and on top of that, there were no negative side effects. However, the 
FDA has yet to approve this new drug, even with such promising results.
  Mr. Speaker, Duchenne muscular dystrophy is an awful condition that 
more or less guarantees a short life for those who live with it. 
Medical advancement is giving hope to those patients for a longer and a 
better life, but bureaucratic redtape is preventing those folks from 
accessing it. It is time for the FDA to remove those roadblocks and to 
move forward for a new, possible, life-improving treatment.

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