Scientific innovation has led to some pretty incredible breakthroughs in modern medicine. And today, we’re going learn about one of those amazing breakthroughs—a gene therapy called ZOLGENSMA® (onasemnogene abeparvovec-xioi) that stops the progression of spinal muscular atrophy, or SMA, with a one-time infusion.
Don’t worry, you don’t have to be a scientist to understand it. Let me show you how it works.
Children with SMA are born with a missing or nonworking SMN1 gene. ZOLGENSMA is an infusion that delivers SMN genes where they’re needed, in a child’s body, to help keep the muscles working as they should.
Now that you know the basics, let’s dive in a little deeper to see how ZOLGENSMA works in the body and how it helps children with SMA.
A dedicated team of scientists at Novartis Gene Therapies approached SMA treatment in a bold way—by targeting the genetic root cause of SMA with a one-time infusion.
Why is targeting the genetic root cause so important? Well, the SMN1 gene provides instructions for motor neuron cells to make SMN protein.
Without enough SMN protein, the motor neuron cells stop working—and that's a big problem!
These motor neuron cells are responsible for all types of muscle movement that we rely on daily for eating, breathing, sitting up, walking, or, when you hear a great song, singing.
If there isn’t enough SMN protein being made, motor neurons stop working. Then, these basic functions we rely on become harder to do and can be lost permanently. As you can imagine, the sooner treatment is received, the sooner the progression of SMA can be stopped.
This is where the amazing science behind ZOLGENSMA comes in. ZOLGENSMA is designed to stop the progression of SMA.
The main components of ZOLGENSMA are a gene and a vector.
The gene is a new, fully functioning SMN gene that's just waiting to get to work.
The vector is made from a type of virus called AAV9, which is not known to make people sick.
The viral DNA is removed and replaced with the new SMN gene, which is then delivered to the right places.
Think of the vector as a delivery truck—with its cargo, in this case the new SMN gene, it’s ready to make its delivery to the motor neuron cells. This process happens throughout the body with many new SMN genes getting delivered to their destination.
That’s pretty cool, right? Even cooler is the fact that more than two-thousand children have received ZOLGENSMA.
Thanks for watching. And if you have any questions about ZOLGENSMA, make sure you talk to your doctor.
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