Mila Makovec with her grandparents, Jeni and Jim Vitarello, in the hospital after receiving a dose of milasen in 2019
The apparent success of milasen is stunning proof of how these designer genetic drugs, known as antisense oligonucleotides, can be personalized for individuals, researchers say. Mila Makovec walks on a beach as a young child before she was diagnosed with Batten disease, a rare genetic disorder. “I realized later that her vision was going," Vitarello says."She was not able to make as much sense of the world around her."
“This was a really unusual case,” he says. Genes are written like a sentence explaining how a protein should be put together. But in Mila’s case, an extra piece of genetic code was thrown into the middle of that sentence, Yu says. “It acts as a molecular patch,” Yu says. The custom genetic drug simply covers up the messed up part of the gene, like crossing out nonsense.
Milasen is far from a cure, however, and Vitarello says while Mila is getting better in some ways, she seems to be getting worse in others. She no longer responds as well or as quickly to her favorite songs and stories, for instance.
commonhealth Not covered by any insurance. No one could afford it.
commonhealth Amazing
commonhealth It’s an incredible development. I wish that these kinds of endeavors is where we focused our abilities as a society - rather than the current fruit of sheer greed. I hope I live to see such a society.
commonhealth Sounds great....til you figure in the amount of privilege and money something like this will take. In other words, if you're from the ghetto, this is a fictional novel
commonhealth Making the science (and art) of medicine extraordinarily personal again- just at it was meant to be...
commonhealth Glad things worked out for them. Also, 2.5 million children in the US are homeless.
commonhealth I’m sure the drug is very affordable too
commonhealth Just wondering if this was a child of color would they have made this drug. I would like to believe so, but experience says no.
commonhealth Guess her family was rich.
commonhealth If she survives. She better fucking discover a new planet or something.
commonhealth Cool
commonhealth That’s wonderful. Myself and probably a few other Americans have used a migraine drug for years that is no longer available because the FDA changed their testing guidelines, and this drug is too old to be grandfathered in. It’s called Midrin.
commonhealth Of note, the development cost over 3 million dollars and does not significantly improve the quality of life of the patient.
commonhealth Wow.🥰
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