Key Points
In early 2017, a neurologist at Boston Children's Hospital called Timothy Yu began work on the most ambitious project of his life: to design and synthesise an experimental drug for a dying child, within a timeframe of just a few months...
"This story is a really powerful example of what's possible," says Richard Scott, chief medical officer at Genomics England, which is run by the UK's department of health, and a consultant at Great Ormond Street Hospital in London.. Mila's story illustrates both the promise of personalised medicine, but also some of its frustrations..
Working from Mila's bedroom, Vitarello spends her time speaking to scientists, regulators, and drug companies around the world in the hope of continuing the progress that was made with her daughter's treatment so it can help others with rare diseases...
A study of 7,000 people published in February found they experienced significantly fewer side effects when the doses of certain drugs were tailored to their DNA, a process called pharmacogenetic testing...
But only if parents agree.. "A lot of the work we'll do through the programme is about understanding people's attitudes to how we might store and hold data, what the expectations are, and how you might empower people to be part of decisions about how their data is used," says Scott, "For example, if a child falls ill two or three years down the line, whether it's useful to hold the data so you can access it more rapidly to make a diagnosis, or whether it might help predict the right doses of a particular medicine later in life..
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