[Congressional Record (Bound Edition), Volume 147 (2001), Part 10]
[Senate]
[Page 14110]
[From the U.S. Government Publishing Office, www.gpo.gov]



                      MUSCULAR DYSTROPHY RESEARCH

  Mr. BURNS. Madam President, S. 805, introduced on May 1, is a vital 
step toward the day when advanced research will find ways to halt, and 
even cure, life-threatening muscular dystrophy.
  Muscular dystrophy is a genetic disorder, actually a number of 
separate disorders, that are characterized by weakening and eventual 
wasting of muscles throughout the body. A quarter of a million 
Americans of all ages are affected by these disorders. One form, 
Duchenne, strikes young boys and usually takes their lives before they 
reach their twenties. Other forms that affect adults are also severely 
debilitating and can be devastating to the victims and their families.
  Since 1966, entertainer Jerry Lewis has hosted the annual Muscular 
Dystrophy Labor Day Telethon, calling the Nation's attention to the 
muscular dystrophies and seeking help for individuals and families 
affected by these diseases. Jerry Lewis is the National Chairman of the 
Muscular Dystrophy Association which, through its Telethon and year-
round fund raising activities, has raised hundreds of millions of 
dollars for programs of direct patient services, research and summer 
camp. The MDA program supports a nationwide network of 230 clinics, 
which are affiliated with hospitals and universities, sends more than 
4,000 youngsters it serves to MDA summer camps, and helps pay for 
wheelchairs, braces, and various therapies for people with muscular 
dystrophy.
  In addition to providing these direct patient and family services, 
MDA expends about $30 million per year to support scientific research. 
Over the past half century, MDA has funded research that was vital in 
developing the protocols that resulted in groundbreaking discoveries in 
genetic mapping. This extraordinary organization has played a key role 
in identifying the gene defects that cause virtually all of the forms 
of muscular dystrophy. The Muscular Dystrophy Association is to be 
commended for its work and can be justifiably proud of the very 
positive role it has in assisting those affected by neuromuscular 
disease. In fact, the implications of their research extend to all of 
the estimated 5,000 genetic-based diseases affecting all of mankind. 
With all of the research insights and opportunities made available by 
this organization, it is time for us to help.
  The next critical phase in muscular dystrophy research is to apply 
these basic scientific discoveries to the development of effective 
therapies. That will require substantial Federal funding. Authorizing 
such a vigorous Federal effort is the purpose of S. 805. The bill calls 
upon NIH and the Centers for Disease Control to establish Centers of 
Excellence in which intensified clinical research can be conducted 
which will speed the discovery of treatments and cures for the various 
forms of muscular dystrophy.
  S. 805 provides the Director of the NIH and the Directors of the 
several institutes within NIH that conduct muscular dystrophy research 
with the authority and responsibility to concentrate and intensify that 
research effort. The bill also authorizes the funds needed to conduct 
essential clinical trials. In short, it gives NIH the organization and 
the mandate to exploit recent advances in gene therapy. The goal is the 
swiftest possible rescue for children and adults whose lives will 
otherwise be lost or badly damaged by muscular dystrophy.
  Mr. President, the Congress has responded generously and often to the 
demands for research funding aimed at other diseases that shorten or 
impair the lives of Americans. It is time to add muscular dystrophy to 
the list of those diseases. I commend my colleagues for introducing S. 
805, and I regret that I am just now getting the opportunity to deliver 
this statement, two weeks after my name was added to this important 
legislation as a cosponsor.

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