Managed Access Agreement resources
For patients and clinicians
Vimizim Treatment for Morquio A – The Managed Access Agreement
Brineura Treatment for CLN2 Disease – The Managed Access Agreement
Brineura® (cerliponase alfa) is an enzyme replacement therapy for the treatment of CLN2 disease (Batten disease). It is the first disease-modifying treatment available for CLN2 disease, which was authorised for use in 2017. It is currently available in England through a Managed Access Agreement (MAA).
This guide helps explain the MAA, the criteria set out in it to allow a child to start treatment with Brineura and how treatment is monitored during the agreement.
This parent/carer guide is available for download in English and will soon be available in Polish.
If you are a patient or parent/carer for someone on one of the above Managed Access Agreements and need more guidance please contact your consultant.
Further support can be offered by your patient organisation, please refer to the final page on the Managed Access Agreement guide for contact details.