A bespoke genetic therapy is helping Susannah. Can similar drugs be made at scale for other rare diseases?

Will it work?

It was the question everyone around Susannah Rosen kept asking. Since infancy, she’d lived with a rare neurologic disease. She’d struggled to walk and talk, only to have the degenerative disease mostly rob her of those skills. Hardly a day passed without her suffering seizures, leaving her in need of constant care.

But in October 2022, Susannah’s family had reason to be hopeful. Scientists had developed a bespoke treatment for her, a snippet of genetic code targeting the gene at the heart of those symptoms. And a doctor had just injected this therapy into the 8-year-old’s spine. From there it would wend its way to her brain.

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