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Mila’s Legacy

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How many medicines can you think of created for just one person? The likelihood is none - which is why the world hasn’t heard of milasen yet. But its creation, and the efforts behind it, could build a pathway towards some of the greatest advances in genomic medicine, and a new initiative being trialled in Britain has a huge role to play in making this happen. At the age of seven, Mila Makovec became the first person in the world to be treated with a medicine created just for her. A bubbly young girl from Colorado, Mila suffered from a very rare genetic disorder called Batten disease, which leads to a painful early death in children. Mila’s mother, Julia Vitarello, resolutely sought out scientists to try to discover a way to save her daughter. After relentless efforts, one doctor, Timothy Yu from Boston Children’s Hospital, imagined a possible treatment for Mila. The challenge was it involved making a completely unique treatment for Mila’s specific genetic mutation. It would be novel and very expensive -