A prime FDA official weighs a way forward for individualized therapies

Again in 2016, a 6-year-old named Mila was identified with Batten illness, a progressive and incurable genetic syndrome that will brutally strip away her sight and her capacity to stroll, and would trigger dozens of seizures every day. The situation is deadly.

However her mother and father, Julia Vitarello and Alek Makovec, refused to lose hope. They contacted Dr. Tim Yu, a neurologist at Boston Youngsters’s Hospital, who found the exact genetic mutation that precipitated her illness — and was capable of devise an experimental remedy designed solely for Mila. His work is among the first examples of therapies created for the advantage of a single affected person — an “N of 1” examine.

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