Our condition costs millions, our lives are priceless. Treat SMA!
Today is SMA Awareness Day.
An SMA gene therapy trial starting in London
We are excited to share the news that a clinical trial of AVXS-101, the first experimental genetic treatment for spinal muscular atrophy, commenced in London this week.
Registration open for next NICE Committee meeting on Spinraza
Registration is now open for the NICE meeting on Spinraza which will be held at NICE Manchester office on Tuesday 23 October 2018.
At a Spinraza meeting at NICE today
Today NICE held a closed-door meeting dedicated to discussing challenges with nusinersen appraisal. Clinical experts as well as TreatSMA, SMA Support UK and MD UK were invited to present their views.
Prime Minister asked about Spinraza
During yesterday’s Questions Time, Mary Glindon MP asked the Prime Minister about access to Spinraza.
TreatSMA advocates for universal newborn screening for SMA
This week we submitted our comments to the NHS consultation on newborn screening for spinal muscular atrophy. We were shocked to learn that NHS argues against introducing this procedure, and we strongly objected to their views.
TreatSMA has responded to draft nusinersen guidance
We have submitted our comments to the draft NICE guidance on nusinersen.
SMA community rallies nationwide to demand access to Spinraza treatment
Yesterday, we stood demanding the right for the SMA community to access the only treatment, Spinraza, for this devastating condition that currently holds the title of the biggest genetic killer of children under 2.