General: 07354 486 586   |   Fundraising: 07511 821 181   |   Support: 0800 046 9832  

Life-changing drug for rare and fatal form of childhood dementia made available in Scotland for the first time

Today, the Scottish Medicines Consortium has recommended the use of Brineura® (cerliponase alfa) in the NHS to treat CLN2, a form of Batten disease.

Batten disease is an ultra-rare inherited degenerative condition that robs children of their eyesight, speech and mobility. It is part of a group of conditions for which childhood dementia is one of the symptoms.

Brineura® is the first and only treatment of its kind to improve the lives of children affected by this life-threatening condition. Its approval in Scotland provides new hope for patients who have no other treatment options. Brineura® has been shown to slow the progression of the disease and give better outcomes for children with CLN2 Batten disease. The younger a child can access Brineura®, the better the outcome.

Without treatment, children do not survive to adulthood. It is currently estimated that there are between 30-40 children in the UK with the condition, though more are believed to suffer from the disease without a diagnosis. There are 13 types of Batten disease and CLN2 is one of the most common.

Low awareness of the signs and symptoms among patients and limited NHS guidance to inform doctors about the disease means that cases are often identified late, when children are already severely disabled. It is hoped that, if NHS Scotland can work to improve clinical awareness of the signs and symptoms of Batten disease, this treatment will be able to help more children.

Brineura® is already available to eligible patients in England and Wales. Brineura® is one of the first medicines to be assessed under a new Scottish ultra-orphan medicine pathway for appraising ultra-rare treatments. Once a treatment goes through this pathway, it can be made available through the NHS in Scotland for a period of three years prior to a decision on further routine use. This means that children and their families do not have to face long delays to access potentially life-saving medicines.  

Michelle Curran, mother of five-year-old Violet who lives with CLN2 and has been receiving treatment with Brineura®, welcomed the news, saying “Nothing prepares you for the news of your child being diagnosed with Batten disease. For us, Brineura has given us hope for the future, that so many families before us have not had. Since Violet has been receiving treatment, we have been blessed with skills returning that she had previously lost, mobility improving and parts of her personality creeping in again. We see Brineura as a miracle. It has given us the possibility of a future with our beautiful daughter.”

Amanda Mortensen, Chief Executive of the Batten Disease Family Association said, “Today is a life-changing day for families in Scotland who are affected by this tragic illness and those families who will be affected in the future. We are so pleased that the Scottish Government has joined England and Wales in making the first ever treatment for CLN2 available to patients. The process in Scotland is also welcome; in England it took several years for patients receive treatment, resulting in patients deteriorating and families losing hope.”

Make a donation:

To make a donation please click the button to the right.  This link will then take you to a PayPal page where a donation direct to us can be made.

The amount you enter is your choice and PayPal can collect the funds from a debit or credit card account.

Thank you for your donation.