They Created a Drug for Susannah. What About Millions of Other Patients?

December 19, 2022

(New York Times) – Susannah was the first person to receive a drug designed to treat KIF1A-associated neurological disorder, or KAND, a progressive disease caused by genetic mutations that affect just 400 people in the world. In doing so, the young girl and her parents have found themselves on the edge of personalized medicine. Since the technology for such bespoke genetic drugs debuted in 2018, about two dozen patients have received the infusions — costing as much as $2 million per patient — to treat a range of neurological syndromes. But hundreds of millions of others, mostly children, live with rare genetic diseases and have no treatment options. (Read More)