Please amend the letter below to suit your circumstances and include anything you would like to say. If you do not know who your MP is then you can check by visiting: https://www.parliament.uk/mps-lords-and-offices/mps/
Dear NAME OF MP
Today is Rare Disease Day, a day when all those with rare diseases, their families and friends celebrate their diversity and the contributions those with rare conditions make to our communities. Sadly, for families of children with CLN2 Batten disease there is no cause for celebration as Brineura, the only treatment for this or any other type of Batten disease, was not recommended by NICE last Friday.
This means children will be denied the treatment they need to prolong their lives.
Batten disease is a form of childhood dementia. CLN2 (Late Infantile Batten disease) is a rare genetic neurodegenerative disease, 5-6 children are diagnosed with this disease each year. Children are born seemingly healthy and develop normally for the first few years of life. Towards the age of 2 their development may slow, including their ability to talk. The first definite sign of the disease is seizures, may be drops, vacant spells or motor seizures with violent jerking of the limbs and loss of consciousness. Seizures may be controlled by medicines for several months but always recur, becoming difficult to control. Children become unsteady on their feet with frequent falls and gradually skills such as walking, playing and speech are lost. By 4-5 years, the children usually have movement disorders such as myoclonic jerks and dystonia. Children with CLN2 disease develop childhood dementia and their short term memory is lost. Vision is also gradually lost and children become completely blind. By the age of 6 years, most will be completely dependent on families and carers for all of their daily needs. Death usually occurs between the ages of 6 and 12 years.
The treatment was not recommended because of uncertainty, a lack of long-term evidence and price. It has, however, been approved and patients have access to it in 20 other countries, including Croatia, France, Germany and Wales.
I have been following the progress of Brineura through the NICE process, learning more about the challenges faced by the families of children with Batten disease and how loved, cherished and valued these children are by their families. I cannot imagine the grief and fear their families must be feeling. I am deeply concerned. I ask that you show your support by asking the Secretary of State for Health and Social Care to mark Rare Disease Day by committing to meeting with the families impacted by this decision and the Batten Disease Family Association.
My family are/have been in this situation with Batten disease/another rare disease so understand the challenges the families are facing. I cannot imagine having the hope that this treatment offers snatched away like this. I am deeply concerned. I ask that you show your support asking the Secretary of State for Health and Social Care to mark Rare Disease Day by committing to meeting with the families impacted by this decision and the Batten Disease Family Association.
I look forward to hearing from you.