Treatment of a teenager with an ultra-rare condition is a medical milestone
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.
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