Scientists really help patients and I
really thought that science was the key
to helping people. My whole life has been
devoted to that.
Muscular dystrophy is a degenerative
disease of muscle. The first gene for
muscular dystrophy was discovered in
1987. Duchenne was the most common and
the most severe of the muscle diseases. I
mean it was really devastating and
seeing how many patients died of the
disease and it was really a hopeless
feeling. The Center for gene therapy was
very pioneering. I wanted everybody in
the center to be focused and targeted on
neuromuscular diseases. I'm a clinician
scientist primarily but have roots also
in the lab and could show a lot of the
basic scientists how we move from the
lab to the clinic. Gene therapy is better
than any drug therapy because its a
one-time delivery. If we treat patients
very early we can get to the disease
before it wipes out all of the muscle or
nerve. We've done more gene therapy
trials than any place in the world. In
muscle disease. We've had more successes
than any place else in the world in
muscle disease. That's what I've been
doing for the last forty nine years and
I'd like to leave this actually curing
these diseases and I think that's on the
horizon.
