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Finally, the news we had fought so hard for arrived in September 2019 — Brineura (Cerliponase Alfa) was approved for funding by the NHS.

Ollie and Amelia are now among 23 children throughout England who will finally benefit from the recommendation made by the National Institute for Care and Excellence (NICE) in September 2019 to include Brineura in the National Health Service (NHS).

What treatment is available (on the NHS) for Batten disease?

Infants with CLN2 start showing symptoms of the disease between ages 2 and 4, lose their ability to walk by late childhood, and usually don’t survive past their teens. Thanks to this specialist treatment, the effects of the disease on our two children have been halted.

Brineura was approved for use by CLN2 patients across the European Union in June 2017, but wasn’t included in national health systems that cover the costs. Historically, the National Institute for Health and Care Excellence concluded that Brineura was not a cost-effective NHS treatment option for patients of Batten disease.

But now, with Brineura available on the NHS, it could boost the life expectancy of all affected children in England from 13 years to 60.

What does it mean for Ollie and Amelia?

In 2016, Ollie was granted compassionate use of the drug directly from the American pharmaceutical company, Biomarin. But with the drug company only able to provide the treatment on an indefinite basis, we pinned our hopes on the NHS.

This news gives us comfort and peace of mind that both Ollie and Amelia will receive the treatment.

Although Brineura is now available on the NHS, it is only available until 2024 under a five year managed access agreement. At this point, NICE and NHSE will review the data to decide whether to continue funding the treatment or not. We continue our fight to prove that this drug is beneficial to children battling from CNL2 Batten Disease.

Even though Ollie may never be able to walk or talk, his quality of life has improved massively. We even managed to go on a family holiday to Disney World in Florida. That would never have been possible before.

Amelia started the treatment before her symptoms emerged and is able to attend mainstream school and enjoy dance, gymnastics and swimming. We hope that the NHS, NICE and BioMarin will work together to ensure that all children with Batten disease in this country will have access to this life-saving treatment.

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