Translarna - January 2021 update
5th January 2021
NICE have scheduled a re-review of Translarna to be completed in July 2021. Translarna has been available to some Duchenne patients since 2016 through a Managed Access Agreement and the re-review was expected to be completed over the coming months. This re-review has been delayed due to COVID-19 and we are aware that NICE is unable to provide further details about when it will take place. We would like to reassure patients and their families that this delay should not result in a disruption to treatment with Translarna.
We will continue to keep you updated as we have more news and will prepare to share with NICE evidence generated from Project HERCULES to help inform the re-review of Translarna.
For further information, please contact [email protected].
NOTES FOR EDITORS
What is Duchenne Muscular Dystrophy?
Duchenne muscular dystrophy (DMD) is the most common fatal genetic disease diagnosed in childhood. Children born with DMD cannot produce the protein dystrophin which is vital for muscle strength and function. Muscle weakness starts in early childhood. Many use a wheelchair by around the age of 12. As deterioration continues it leads to paralysis and early death, often in their 20s. It almost exclusively affects boys. There is no treatment or cure. In the UK there are around 2,500 boys affected and around 300, 000 worldwide. It is classified as a rare disease.
Who are Duchenne UK?
Duchenne UK has one clear aim – to end Duchenne, a devastating muscle-wasting disease. As the leading Duchenne charity in the UK, we connect the best researchers with industry, the NHS and families to challenge every stage of drug development to make the incurable, curable. Together, we will find treatments and cures for this generation of patients with Duchenne.
Our president is HRH The Duchess of Cornwall. Our patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell.
We need your help, because we need to keep funding promising new research.
How to donate?
Duchenne UK is entirely reliant on donations to fund research for treatments and a cure to DMD. This can be done via:
- Direct Debit – Duchenne Direct
- Individual Donation – Donate
- If you are a family or friend affected by DMD you can set up your own fund with Duchenne UK – Family and Friend Funds
- Take part in one of our fundraising events – Events
- Text DUCHENNE to 70085 to donate £5. This costs £5 plus a std rate msg.
For more information visit www.duchenneuk.org
Published on 5 January 2021Share this article Categories Policy and campaigning