[Congressional Record Volume 147, Number 103 (Monday, July 23, 2001)]
[Senate]
[Page S8056]
From the Congressional Record Online through the 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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