Baby Among the First to Receive Gene Therapy for Spinal Muscular Atrophy After FDA Approval

Baby Among the First to Receive Gene Therapy for Spinal Muscular Atrophy After FDA Approval


He doesn’t know it yet but baby Iverson
is making history today. We’ve waited so long for this moment. It means everything. Iverson is among the first to get gene therapy for spinal muscular atrophy
since it was approved by the FDA. The gene transfer is given one time and the
gene will begin to produce a protein that this baby is missing. It’s one
little protein. (We’re all set to go and treat.) but you need it in order to be
able to smile, to suck, to swallow, to bend your arms, to move your legs.
When my son rolls over he has to fight for it and like as a dad that kills me.
He lost his ability to eat. He wasn’t able to move like normal babies. He couldn’t cry.
He was on a breathing tube. And then we found out he had SMA and I never even
heard of that. I didn’t even know what that meant. To even be a candidate for
the gene therapy it’s just a blessing. The work that led to this was done right
here at Nationwide Children’s Hospital. We’ve been doing research with this
product from the very first patient that was ever treated in the world. Dr. Jerry
Mendell led these efforts to first prove this would work in animal models and
then did the preliminary phase one studies in children and demonstrated
that this was safe and at the right effective dose. It set us up to be able
to move this forward to all children. I’m just so glad that he fought for
this and I will continue fighting for him. Hopefully we just start seeing
better results. I can’t wait to be able to see my buddy crawl, move, hopefully I
got my fingers crossed on that. A milestone that will certainly make baby
Iverson a history-maker in the world of gene therapy.

One Reply to “Baby Among the First to Receive Gene Therapy for Spinal Muscular Atrophy After FDA Approval”

Add a Comment

Your email address will not be published. Required fields are marked *