[Congressional Record (Bound Edition), Volume 162 (2016), Part 12]
[Extensions of Remarks]
[Page 15919]
[From the U.S. Government Publishing Office, www.gpo.gov]




                PROVIDING CARE TO THE DUCHENNE COMMUNITY

                                 ______
                                 

                        HON. COLLIN C. PETERSON

                              of minnesota

                    in the house of representatives

                       Tuesday, December 6, 2016

  Mr. PETERSON. Mr. Speaker, today I would like to recall the 
significance of the authority extended by Congress to the Food and Drug 
Administration in the 2012 Food and Drug Administration Safety and 
Innovation Act. This legislation enhanced the FDA's ability to develop 
and implement accelerated approval programs to provide therapies to 
patients with rare, debilitating, and one-hundred percent fatal 
diseases.
  Duchenne muscular dystrophy is a genetic disorder resulting in 
progressive muscle degeneration. This rare condition, which typically 
affects boys, is characterized by lung and heart complications that 
cause certain premature death.
  Despite compelling evidence of its efficacy, the FDA recently refused 
to consider a New Drug Application for a therapy developed to treat a 
commonly diagnosed form of Duchenne.
  As there are no alternative treatments for this particular form of 
Duchenne eligible for purchase or approval, Duchenne patients are left 
unable to mitigate the effects of the deadly disease.
  I urge the FDA to grant a full and fair review to New Drug 
Applications for Duchenne therapies in accordance with the Food and 
Drug Administration Safety and Innovation Act. The timely delivery of 
treatments is of the utmost importance to the Duchenne community.

                          ____________________