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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