Illustration credit: Molly Ferguson

Fam­i­lies raised mil­lions and hand­ed rare dis­ease ther­a­pies off to biotechs. But com­pa­nies have backed out

When Ju­lia Tar­avel­la’s two sons were di­ag­nosed with an ul­tra-rare dis­ease, she did some­thing ex­tra­or­di­nary but not un­com­mon among par­ents like her: She formed a group that raised $1.2 mil­lion to try and find a treat­ment.

“With­out a ther­a­py, my sons will die,” Tar­avel­la said.

The mon­ey helped launch work on a po­ten­tial gene ther­a­py at the Uni­ver­si­ty of North Car­oli­na, which was then hand­ed off to a New York-based com­pa­ny called Neu­ro­gene.

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