Gene Therapy Breakthrough for SMA

DR. KUNTZ: Today
has been a very, very exciting day for the
neuromuscular research team here at Lurie Children’s. DR. RAO: This is
a really big occasion for spinal muscular
atrophy treatment, but also a really big
occasion for medicine and medical
advancements in general. ZION’S DAD: It’s a second
chance at life for him. ZION’S MOM: It feels like you’re
in this tunnel for a long time, and you just felt like it
was only going to get worse. MIKAELA’S DAD: This is a big
day for us and our family. She was diagnosed with spinal
muscular atrophy type 1. So it was a initial prognosis
of six months to a year. And fortunately, due to new
medication being on the market, she’s been able to give
this genetic disorder a heck of a fight. MIKAELA’S MOM: When we
got the news, I said, do you want that gene
you’re missing, buddy? And she nodded her head. And I said, all right,
let’s go get it. DR. RAO: So spinal muscular
atrophy, especially type 1 SMA, was universally fatal. And six years ago, we
were offering treatments that were mainly supportive. And most of these children
sadly used to pass away by a year or 18 months of age. MIKAELA’S MOM: We knew we had
a huge team in our corner. She has so many people
pulling for her, and I think it starts
with Dr. Rao and his team. ZION’S MOM: We felt like
just everything was ending. I just can’t even tell you
how low and how dark we felt. But having him just
have so much hope and positivity, it kind
of forced us to think, OK, we can still
keep moving forward. DR. RAO: There’s a
lot of hope for children with spinal muscular atrophy. We went from no
treatments to a treatment that works with such
efficacy that we had never seen in medicine before. And we went from children
dying to now children not only living but
achieving milestones. So it’s almost
like a zero to 60– you know, that quickly.

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