Treatments for rare diseases are more feasible, but access remains limited

Will it work?

It was the question everyone around Susannah Rosen kept asking. Since childhood she suffered from a rare neurological disease. She had difficulty walking and talking, but the degenerative disease robbed her of most of those skills. Barely a day went by without her having seizures, requiring constant care.

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