
Julia Vitarello’s daughter Mila received a first of its kind, custom-built medicine in 2018, programmed to counter the specific mutation that caused the once adventurous young girl to decline rapidly. The treatment, while seemingly helpful, came too late to save Mila, who died in 2021. But Vitarello has since dedicated her life to making such medicines broadly available to potentially millions of children with ultra-rare mutations, founding an international collective of researchers and a biotech, igniting a U.K. government effort, and trying to push the same in the U.S.
From STAT:
A mother, shaped by tragedy, embarks on a mission to advance custom medicines