It will change regulators’ rule books
Mila Makovec was ten in 2021 when she died from an ultra-rare neurodegenerative disorder. But, though it had not saved her, she was nevertheless in the history books as the first to receive a drug designed for a single patient. And her story did not end there. After her death her mother, Julia Vitarello, set out to change how drugs are made, so that others with obscure genetic faults could have bespoke treatments more quickly and easily.
Ultra-rare disorders are those affecting fewer than one person in 50,000. Sometimes, they are unique. And unique problems need unique solutions. To that end, Ms Vitarello had, when her daughter was diagnosed, launched Mila’s Miracle Foundation with the goal of finding her a treatment and paying for it. In collaboration with doctors at Boston Children’s Hospital, it did so in the form of a molecule called an antisense oligonucleotide (ASO).
This article appeared in the Science & technology section of the print edition under the headline “Custom cures”
From the January 24th 2026 edition
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