foreign [Music]
He was five at the time the doctor looked at him and immediately knew he had Duchenne Muscular
Dystrophy. We didn't know anything about Duchenne Muscular Dystrophy. It was very scary to think
what Easton's future might look like. [Music] Muscular dystrophy is a severe life-threatening
and devastating disease. It begins at birth and progresses from that point forward. First,
weakness of the lower extremities. By the time kids get to be teenagers they're stuck
in a wheelchair. Then shoulder weakness, problems lifting arms over the head or
reaching for things. Then respiratory and cardiac involvement and then early death.
The heart really is the end game for these boys.[Music]
Connor was the first patient we ever did with systemic gene therapy. Easton
was the oldest boy in that trial. We have programmed the virus to be delivered over
an hour and 15 minutes. It's not that long but during the viral delivery we exercise
the extremities every 10 minutes. We do the shoulders, the elbows, the wrists, the hands,
the knees, the hips so we can make sure that the virus is distributed over all the muscles.
After the gene therapy, it's like a new child. He had this confidence that he wanted to try
stairs and he wanted to walk around when we were out and about and he wanted to be
more independent. And we still see that to this day. The most remarkable thing was he'd
start just cruising up those stairs and that's nothing that we ever saw him do before [Music]
Almost unbelievable I've been working on this for 50 years and when I saw the first
patient and his disability I felt committed to making a change. Easton just had his five-year
follow-up and your leg right here holding now all of a sudden went from cars playing
on the floor to airplanes. Everything was just cruising around the house it was like he felt
too good to sit down. This therapy gave Easton his childhood and it let him be a little boy.
I think when they signed up for this they didn't know what the outcome would be but now they have
to feel this great sense of gratification for their participation they have helped basically
thousands of boys with duchenne muscular dystrophy. With the gene therapy we were
given that hope back we were able to visualize his future and what he could become and everything
that he could be great at. I hope that with the gene therapy getting approved that opens the
doors for many other families I don't think that he looks at himself as a true Trailblazer but we
tell him all the time he's done remarkable things and what he's doing is helping boys everywhere.