A Treatment for Zoe: Inside the Race to Build a Therapy for a Devastating Rare Disease

January 3, 2017

(STAT News) -The Sanfilippo story is a window into the fraught world of rare disease research. Drug companies increasingly see opportunity in the field, both to alleviate suffering and to reap huge profits. Regulators are under enormous pressure to approve new therapies even with scant evidence that they’re effective. And families seize on every risky trial as a last chance to save a beloved child.