A mum in Coventry faces an agonising wait as Batten Disease funding remains in limbo
A public meeting was held yesterday to discuss future funding for Batten Disease treatment
Last updated 4th Apr 2025
A mum from Coventry continues to face an agonising wait over the future of her daughter's Batten Disease treatment.
Six year old Florence suffers from Batten Disease which causes seizures, sight loss and takes away a child's ability to walk and talk. There is currently no cure for Batten Disease but children, like Florence, are placed on an enzyme replacement therapy which slows the disease down.
The treatment began in 2019 based on a five-year agreement, whilst the NHS collected long term data on how effective it was. That agreement was extended until May 2025 and now commercial discussions are taking place between NHS England and the pharmaceutical company behind it.
We've been speaking with Florence's mum Phoebe on the current state of limbo parents of Batten Disease continue to face as the waiting game over treatment continues.
Phoebe says: "Florence is on a treatment which she started as soon as she was diagnosed at the age of three. Now Florence is six years old and she is still walking, still running, still climbing, still eating, still seeing. Her quality of life is absolutely incomparable to the natural history of this disease.
"She is living a wonderful life at six years old. She's going swimming. she's going to school, she's running, she's reading books.
"She's just she's just a little 6 year old girl and to have this treatment removed would be catastrophic for her. It would cause her to rapidly deteriorate and a premature death would be inevitable."
Phoebe continues: "Living with this anticipatory grief and this constant worry that the treatment could be taken away is quite simply, torture.
"We know that this is working. The main thing for us at the moment is trying to keep her healthy and stable and with us for as long as possible.
"Living with this over our heads constantly worrying about whether NICE are going to approve it and allow children to stay on it, but also future children that are diagnosed to start it, is just absolutely agonising."
The decision on whether to continue funding the drug is now with NHS England, which is currently in commercial discussions with the US pharmaceutical company which makes the drug.
A NICE spokesperson said: "With the support of NICE, NHS England and BioMarin have an agreement in principle for an additional 3-month extension of the current managed access agreement. This will now run until 27 Aug 2025 under the same terms as the previous extension to ensure continued access for current and new patients until our evaluation is concluded."