STAT+: FDA approves a new gene therapy for Sanfilippo syndrome, an ultra-rare disease

The Food and Drug Administration on Thursday approved a new gene therapy for Sanfilippo syndrome type A, an ultra-rare disease sometimes known as childhood Alzheimer’s. The therapy, called Fayuvi, was developed by Ultragenyx. It is the first drug specifically approved to treat Sanfilippo. Ultragenyx did not immediately release a price. “It’s hard to overstate what this approval would mean for everybody who is living with this really horrific disease and watching their children suffer and pass aw...