Every penny we raise will be going into research and the clinical trials to help save the lives of children with Sanfilippo. Sanfilippo disease, results in progressive mental decline, severe behavioral difficulties and a greatly shortened lifespan due to storage of these complex sugars in the brain. At present there are approximately 130 individuals in the UK living with Sanfilippo disease A, B and C. With no current treatments available the outlook is bleak. This treatment if successful will be available to both UK and worldwide sufferers today and into the future.

 

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