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In: neurodegenerative genetic disease

Two donors make Ted’s intervention in Poland possible
30 July, 2019

It has been possible yet again, this time with the help of two donors. They would like to remain anonymous as…

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Marta, with Niemann Pick C, fights for a cure with gene therapy
29 July, 2019

Marta is a four and a half year old girl with Niemann Pick C. This is a rare neurodegenerative genetic disease…

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A successful operation in Varsovia
26 June, 2019

Four year old Irai had a rare disease that only affected around 130 children worldwide: the aromatic amino acids de carboxylase…

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