New data has shown that nearly three in four babies born with spinal muscular atrophy (SMA) are now surviving for two years or more thanks to advances in treatment.

Each year in the UK, around 70 children are born with the disease, which causes muscle weakness, progressive loss of movement and paralysis.

Previous studies in the US have shown that that before the availability of treatments, fewer than one in ten (8%) children born with SMA type 1 (SMA1) – the most common form of the condition – survived to the age of 20 months without permanent ventilatory support.

However according to new data from the national SMA Research and Clinical Hub (SMA REACH UK) database, now nearly three quarters (73%) of children with SMA1 in the UK are older than two years.

The research has also shown a reduction in deaths from SMA1. There were just 11 deaths in total recorded across the UK between March 2018-March 2023 – compared to around 25 deaths each year in England between 2008-2017.

The stats are being linked to the roll-out of three new SMA drugs on the NHS from 2019.

NHS chief executive, Amanda Pritchard, said: “It’s fantastic to see that more and more children diagnosed with this debilitating condition are living longer and with greater independence, thanks to cutting-edge treatments now available on the NHS.

“Before 2019 there were no effective drugs for this condition – and, while there is so much still to do, we’re delighted that access to these new and transformative treatments through the NHS is already making a real difference for families, enabling more babies to stand up and take steps.

“This is yet another example of the NHS leading the way in securing access to innovative treatments for those with rare genetic conditions while delivering value for the taxpayer – and with the expertise of our phenomenal paediatricians and wider specialists, we hope that outcomes for children born with SMA will continue to improve in the coming years.”

Image by Rainer Maiores from Pixabay