Translarna (also known as ataluren) is a treatment for people with Duchenne muscular dystrophy with a nonsense genetic mutation, manufactured by pharmaceutical company PTC Therapeutics.
Translarna
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History
In February 2023 NICE recommended it as a treatment for children aged 2 and over with Duchenne muscular, meaning that it is available on the NHS in England, Wales and Northern Ireland. We acted as a patient expert in the appraisal process and presented the views of the Duchenne community after wide consultation and engagement.
The full NICE guidance can be read here.
What we are doing now
In Scotland, Translarna is available through the Scottish Medicines Consortium ultra-orphan pathway until 2025, when it will be reassessed. You can read more about this here.
In the UK, Translarna has a conditional licence from the Medicines and Healthcare products Regulatory Agency (MHRA), which means there is an obligation to confirm effectiveness as more data becomes available. This has been subject to annual renewal but completion of a study called STRIDE means that there is now ‘confirmatory data’ which PTC Therapeutics has submitted for full assessment. This is now being examined by the MHRA and will be discussed by the Commission on Human Medicines (CHM) in late May 2024. We will be part of that meeting to present the views and experiences of people who have received the treatment.
Support and information
Duchenne muscular dystrophy
Duchenne muscular dystrophy is a muscle wasting condition caused by the lack of a protein called dystrophin. It usually affects only boys.
Research
We fund groundbreaking research to learn more about muscle wasting conditions and lead us to new treatments. We’ve already made advances that would have been unthinkable just 10 years ago, and we are determined to go even further and faster.
Our support services
We are here for everyone, but we understand that support isn’t one-size-fits-all. Speak to us. We can tailor our support to meet your unique needs.
Our helpline is open Monday-Thursday 10am-2pm.