Translarna not accepted for routine use on the NHS in Scotland

8 June 2026

Translarna, a treatment for a specific genetic type of Duchenne muscular dystrophy, has not been accepted for routine use on the NHS in Scotland. The treatment had been made available for a limited time to collect more evidence, but the additional data did not show enough benefit to justify the cost.

Translarna (also known as ataluren) is designed to target the genetic cause of a specific type of Duchenne muscular dystrophy (DMD), known as a nonsense mutation. This is one type of genetic change that leads to a lack of dystrophin, a protein that muscles need to work properly. It affects around 10–15% of all DMD cases. 

The Scottish Medicines Consortium (SMC) reached the decision after reviewing the available evidence and the cost of the treatment. They concluded it did not show enough benefit and was not strong enough to support its routine use on the NHS. 

Why this decision was made

We know that decisions like this are difficult to hear, especially when the treatment has been available for a period of time. We’ve heard from families who have told us that having access to Translarna was important for them. For some, it has been about having something to try. For others, it has been about choice. 

At the same time, fully understanding the benefit of a treatment, especially in rare conditions, isn’t always straightforward. Evidence is often limited, and it can take time to build a clearer picture of how well a treatment works in practice. 

That was the situation when Translarna was first introduced. At the time, there were very limited treatment options available, and there was a need to better understand whether it could make a difference in practice. Making the treatment available for a period of time allowed more information to be gathered. 

However, this approach can also create uncertainty for families, as future access to a treatment may change depending on what is learned. This is what we’ve seen with Translarna. After more data was collected, the overall evidence was not considered strong enough to show a benefit that would justify the cost to the NHS in Scotland. 

What does this mean for families in Scotland?

Anyone who is currently receiving Translarna will still be able to access the treatment. However, new families will not be able to access it routinely through the NHS.  

In practice, this is unlikely to affect many families in Scotland, as there have been no new diagnoses of this specific type of DMD for around a decade. However, we recognise this may still be disappointing for families in the future. 

There are also processes in Scotland where NHS boards can consider individual requests if a doctor believes a treatment would be appropriate. 

Several treatments are also now available for DMD, with two new treatments approved for use in Scotland in the last 18 months – vamorolone and givinostat – which may be options for some families.

What about the rest of the UK?

Translarna is currently available for routine use in England, Wales and Northern Ireland. 

Decisions about NHS treatments are made separately across the UK. In Scotland, this is done by the Scottish Medicines Consortium (SMC), while in England it is the responsibility of the National Institute for Health and Care Excellence (NICE). Wales and Northern Ireland generally follow NICE decisions. 

Although these organisations often look at the same evidence, they can reach different decisions because they use slightly different approaches to assess treatments. 

This can lead to a postcode lottery, where access to treatments varies depending on where someone lives. We know this can feel confusing and frustrating for families, particularly when the same evidence is being considered. 

This also reflects the challenges of making decisions in rare conditions, where evidence can be limited and difficult to interpret. Different decision-makers may take different views on what level of evidence is enough, and whether a treatment offers enough benefit for their limited NHS resources. 

We will continue to highlight where differences in access risk creating unfairness for families across the UK, while supporting decision-making that is clear, transparent and based on the best available evidence.