TreatSMA Vigil 6th March 2019
On 6th March, TreatSMA along with many families and adults from the SMA community will once again travel to Manchester for the third appraisal meeting of nusinersen.
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
The TreatSMA team will take part in this year's Rare Disease Day celebrations to build awareness of SMA.
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.