NICE started appraisal procedure for SMA gene therapy
The National Institute of Health and Care Excellence has begun procedures leading to appraisal of AVXS-101, the first gene therapy to treat spinal muscular atrophy.
Biogen Nurture study reveals new and positive data
Nurture Study reveals new and positive data showing benefits of treating pre-symptomatic infants with Spinraza at the annual congress of the World Muscle Society.
Roche announces positive interim results of risdiplam trials
Roche today announced interim clinical data from the dose-finding parts of the pivotal FIREFISH and SUNFISH studies investigating risdiplam as a treatment for spinal muscular atrophy. The data were presented yesterday at the 23rd International Annual Congress of the World Muscle Society in Mendoza, Argentina.
TreatSMA met with the Roche UK team
At the request from TreatSMA, on Monday 24 September five members of TreatSMA held a meeting with Roche in London. We discussed our advocacy work and general wider issues facing the SMA Community.
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.