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 Texas-based company called Neurogene.

But what sounds like a victory propelled by a parent’s perseverance is being overshadowed by a lesserknown but increasingly common trend: Drugmakers are shelving many of those family-funded therapies.

In 2022, almost four years after Neurogene took over, the company stopped the work. The program is back with the University of North Carolina, and Taravella and other parents are trying to raise another $2.3 million for a clinical trial.

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