Today is SMA Awareness Day.
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 is now open for the NICE meeting on Spinraza which will be held at NICE Manchester office on Tuesday 23 October 2018.
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.
During yesterday’s Questions Time, Mary Glindon MP asked the Prime Minister about access to Spinraza.
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.
We have submitted our comments to the draft NICE guidance on nusinersen.
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.