NICE prepares to review intrathecal administration of gene therapy for SMA

As the National Institute for Health and Care Excellence prepares to review intrathecal administration of gene therapy for spinal muscular atrophy (SMA), one of our trustees will be there bringing your voices, your experiences, and your reality into the room.

Because TreatSMA has always been about the community.

Gene therapy such as onasemnogene abeparvovec has already changed lives as a one-time intravenous treatment for young children. Now, the intrathecal approach delivered directly into the spinal fluid is being explored to:

* Open the door for older children and adults

* Reach the central nervous system more directly

* Expand access beyond current limitations

Early studies suggest this could stabilise or improve motor function for those who currently have fewer options, building on progress made with treatments like nusinersen and risdiplam.

But NICE doesn’t just look at data.

They look at:

* How treatments work in real life

* The balance of benefit and safety

* Long-term value for the NHS

* And importantly, the voices of the community living this every day.

That’s why TreatSMA having a seat at the table matters.

Our mission to secure treatment does not stop when the cameras stop rolling. As this review progresses, we may ask to share more of your stories, your perspectives, and your realities with NICE.

Because decisions like this should never be made without the people they affect.

After all, community is what matters.