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At 16 months, Nicole Collicoat (born 2nd March 2004) has exceeded her life expectancy. She has Pompe’s disease. Her sister, Pauline, died of the same disease eight years ago at 14 months. More than 17 years ago her eldest sibling was still-born, now considered to have suffered the same disease. Nicole has exceeded her life expectancy because she’s one of about 17 babies in the world receiving a new and still unlicensed treatment for Pompe’s. The treatment with a drug called Myozyme is currently unavailable to most sufferers because worldwide, Myozyme is licensing approval. Myozyme uses cutting-edge medical technology, and like many drug developments, has its history of politics. And because Pompe’s is a rare disease, the development of this potential cure has been an uncompromising battle of persistence by many: sufferers like International Pompe’s Association (IPA) Board Member and Australian Pompe’s Association (APA) President, Helen Walker; parents of lost children, like Scottish agricultural scientist, Kevin O’Donnell; and scientists like Dr Arnold Reuser and Dr Ans van der Ploeg, the leading world authorities of Pompe’s disease, in the Netherlands.

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