Appeal to support the Treatment against BATTEN DISEASE - a terminal Neurological condition

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This appeal is made by us as parents on behalf of our 5 years old son, who is our only child,  to gather support in favour a new novel treatment to fight against a rare terminal neurological condition called Batten Disease. This condition has various types with different age of onset of symptoms but invariably all types affect the mental development, early childhood dementia, epilepsy, loss of speech, limb co-ordination, balance problems, loss of vision leading to blindness, swallowing difficulty, breathing problems in final stages leading finally to early age death. Our son was diagnosed with one of the types of Batten Disease (CLN2) in March 2017 when he was just over 4 years. Without this new treatment he would have been bed bound by now, however he is still walking.

Thanks to this treatment, which in the form of an Enzyme infusion given directly into the brain through an access port implanted underneath his scalp. A number of leading EU nations and the USA have already accepted this treatment in their health systems on the basis of current trial results. Our son is currently receiving this treatment as a part of the extended trial on compassionate use and he is significantly benefitting from it. However, the UK government's agency NICE, which looks into setting recommendations and guidelines for any new medical treatments in UK, had turned down the application for recommending to commission this treatment in NHS, siting that this treatment is not cost effective. We are completely devastated with this news and have no where to go, if this treatment is stopped on our son once the trial finishes. We can see the difference in him, his condition is not progressive anymore, he is still walking, he is eating and swallowing without any difficulty, he enjoys his TV programmes, he enjoys school, he loves his story books and his iPAD games, he has a life and we have a life because of him. 

A number of affected families are already fighting against this heartbreaking decision and we as a family would request the community to come forward to support this appeal to gather as many signatures as possible to forward this to to NHS England and NICE to reconsider their decision. 



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