Since late 2016, there’s been a gradual worldwide introduction of drug treatments for SMA, but not all are possible for all children in the UK. This may be due to reasons of safety and / or decisions that have been made by UK regulatory authorities based on clinical and cost effectiveness. In general, both clinical trial and real-world evidence suggests that early treatment is necessary to maximise the potential benefits.
What’s possible for my child?
There are currently three drug treatments available for some, but not all children. For the latest information on which children may have access to these treatments in the UK, please follow the links below but, most importantly, talk to your child’s medical team about what is possible for your child.
What experiences have other families had?
Families sometimes ask about the experiences of other families who have been faced with treatment decisions – what influenced their decision making; if they chose a treatment; what the actual treatment delivery experience was like; what outcomes they have seen.
Many families share their experiences on social media and for some these can be useful. It’s worth bearing in mind, though, that no two families have the same personal circumstances and that no two children are the same in terms of the impact of their SMA, what treatment is most appropriate for them, how they will react and what the outcomes will be.
It’s probably fair to say that, on the whole, it’s families who’ve chosen treatments and seen positive outcomes that post their ‘stories’ publicly, so it may still be worth talking through what you do see on social media with your medical team and if you have any questions.
Page last reviewed / updated: May 2021
Next review due: May 2022