First Baby in England Treated with Zolgensma
01 June 2021
It’s very heartening to see the first child with SMA Type 1 being treated with this ground-breaking gene therapy via the NHS at Evelina Hospital, one of the four infusion Centres set up in England. We are pleased to see this coverage on the BBC today as it helps to raise awareness of SMA and its impact on children and families.
We know that there has been a huge amount of work by clinicians and NHS England to get this programme up and running.
Zolgensma could be potentially lifechanging for some babies and children who have already been diagnosed with SMA, and as we support families through this understandably anxious period, we are doing all we can to ensure that those currently waiting will, as soon as possible, be able to have full discussions with their clinical team to see if this treatment option will also be appropriate for their child.
The earlier Zolgensma is given for SMA the better, with pre-symptomatic treatment shown to give the very best possible outcomes. Early diagnosis and treatment is vital. We need newborn screening for SMA to be introduced in the UK so that our children have this and other drug treatment opportunities for their futures.
For the latest information we have, please see our England Q&As.