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.
Category: Updates
NHS refuses newborn screening programmes for SMA
In an absurd decision, the NHS rejects introducing newborn screening programmes in SMA.
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
MD UK and SMA UK are organising a SMA drop-in event on 25 February. Please invite your MP!
Scotland will offer Spinraza treatment to all people with SMA
BREAKING NEWS: Spinraza will be available in Scotland to all with SMA from April.
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
As NICE Committee will review Spinraza on 6 March 2019, the SMA Community stands united behind our right to treatment.
Replies from the NHS and the Department of Health
Replies from the Department of Health and the NHS to the Community’s open letter