A young girl’s custom gene therapy hints at a framework for tailored rare disease treatments
A Boston Children’s Hospital team has developed a second personalized medicine, called atipeksen, for a young child with a rare genetic disorder.
When Timothy Yu developed milasen, a custom drug for a young girl named Mila with Batten disease, he ignited a spark in the field of personalized medicine. Milasen was the first medicine specifically designed for a single person, and it was developed in just about a year.
In response to milasen, nonprofit organizations have emerged calling for the development of personalized therapies for the estimated 400 million people living with rare diseases worldwide. These medicines, colloquially called “n of 1” therapies, are often made to treat debilitating genetic conditions that are too rare to garner interest from pharmaceutical companies.
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