Eleanor Smith MP asks PM Theresa May about Spinraza
Eleanor Smith MP asked the Prime Minister about access to Spinraza during today's PM Question Time.
I lay by my boy as he died at five months…
Paul and Jeanette narrate their struggle with spinal muscular atrophy in a recent edition of The Mirror.
NHS refuses newborn screening programmes for SMA
Contrary to global developments and expert advice, today the NHS formally refused to back a newborn screening programme for spinal muscular atrophy.
Celebrating Rare Disease Day 2019
In less than two weeks we will start celebrating Rare Disease Day – that annual day when all those who are affected by a rare disorder go out and speak about it. The global rare disease community holds celebrations throughout the world. This year TreatSMA will take part with an online campaign and a face-to-face event in the South East.
Parliamentary “drop in” event dedicated to SMA
An event dedicated to the situation of SMA Families will take place on 25th February in the Parliament. Please invite your MP!
Scotland will offer Spinraza treatment to all people with SMA
We are extremely happy to share the long-awaited news: the Scottish government will be making Spinraza available to all people with spinal muscular atrophy in Scotland.
First presymptomatic baby receives gene therapy in London
A newborn girl became the first child in the UK to receive the ground-breaking gene replacement therapy AVXS-101 presymptomatically.
NICE will review Spinraza again on 6 March
For the third time a drug appraisal committee of the National Institute for Health and Care Excellence will meet to review the nusinersen (Spinraza) dossier. The meeting will take place on the 6 March in Manchester.