Action Duchenne and MDUK have received the following public statement from NICE which we can share with the community:
“The Managed Access Agreement (MAA) for ataluren (Translarna) for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene is scheduled to end in July 2021. NICE, NHS England and NHS Improvement and PTC Therapeutics (the Company) are exploring options to extend the MAA to account for the new timeline for the NICE re-evaluation, which was paused as part of NICE’s COVID-19 workplan prioritisation framework. Discussions are continuing and an update will be issued as soon as the outcome is known.”
We would like to provide re-assurance to you that Action Duchenne and Muscular Dystrophy UK jointly are continually and regularly communicating with NICE on this very sensitive issue for families and will provide further updates as soon as we can.
If you have any questions relating to the MAA, please contact gary@actionduchenne.org
Treasuring each day
