Inside a couple’s quest to pay for an experimental gene therapy to save their children

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Image credit: Jennie Landsman
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“We need your help, we really do,” Gary [Landsman] says, his voice breaking. The Landsmans’ two sons—Benny, then 18 months, and Josh, four months—both have a fatal genetic brain disorder called Canavan disease. Benny, limp on his mother’s lap, is already affected by nerve loss. Josh isn’t yet. But he will be if nothing is done.

Jennie hit Google and started e-mailing scientists. Here’s what she learned: there may be a way to fix the genetic error in the boys’ brains. But the family would have to pay for it themselves.

The Landsmans had discovered gene therapy, technology that uses viruses to add healthy genes to cells with defective ones.

In Florida, a single boy was treated with a Canavan gene therapy in 2017 after his parents paid for the experiment. They did it under an exemption in federal rules called “expanded access,” which can allow unapproved drugs to be offered to specific patients “whose life is immediately threatened.”

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That experiment fell into a gray zone, not quite research and not quite medicine. It is the same pathway the Landsman family is trying to follow.

Their video, posted to Facebook and later GoFundMe, a crowdfunding site, went viral. By now, they’ve been on TV and in People magazine. Eight thousand donors have already given more than $1.5 million.

Read full, original post: Two sick children and a $1.5 million bill: One family’s race for a gene therapy cure

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