Translarna/ataluren is used to treat a version of Duchenne muscular dystrophy that is caused by nonsense mutations. It is currently accessed in England on the NHS under what is known as a Managed Access Agreement (MAA). This agreement between NHS England, NICE, and the drug manufacturer allows patients to receive treatments with promising potential while further is gathered data to strengthen the clinical evidence.
On 13 May 2021 NICE announced that the MAA, which began in 2016, is to be extended from its original end date of July 2021 to January 2023. As patient representatives on the Managed Access Oversight Committee, MDUK and Action Duchenne are working with NICE to relay questions from the Duchenne community to NICE and to publish the answers that they provide in response.
This list will be regularly updated. If you would like any questions added please email firstname.lastname@example.org. MDUK will share all questions with Action Duchenne and we will jointly address them with NICE.
The latest version of the Q&A document can be accessed here.