Doctors say gene medicine tailored to a single person can work, but it still costs millions, reports Erika Check Hayden.
Tragedy strikes: When six-year-old Mila Makovec was diagnosed with a devastating neurological disorder called Batten disease, the happy, active girl had already lost her ability to see, to say most words, and to walk without help. There was no known treatment for her fatal condition.
A treatment of her own: Doctors in Boston created a treatment just for Mila, by finding the genetic cause of her disease and designing a drug to overcome the error. It’s believed to be the first individually tailored treatment of its kind, and it’s been partially successful. Mila has fewer seizures, and can eat pureed foods on her own rather than solely through a tube.
Wider lessons: The question is whether others could benefit from similar hyper-personalized medicines, which could cost several millions of dollars each to produce. And, crucially, who would pay for them? Read the full story here.
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