STAT+: FDA approves a new gene therapy for Sanfilippo syndrome, an ultra-rare disease
The FDA approved a new gene therapy for Sanfilippo syndrome type A, an ultra-rare disease sometimes known as childhood Alzheimer’s.
The Food and Drug Administration announced on Thursday its approval of a groundbreaking gene therapy for Sanfilippo syndrome type A, a rare and devastating disease also known as childhood Alzheimer's. This innovative treatment, dubbed Fayuvi, is the first medication specifically designed to combat Sanfilippo syndrome. Developed by Ultragenyx, the drug's approval marks a significant milestone for patients and families grappling with the life-altering impact of this condition.
Cara O'Neill, the chief science officer of the Cure Sanfilippo Foundation, expressed her enthusiasm, stating that this approval offers newfound hope for those living with the disease and their loved ones. "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 away early, and all the rest that it brings," O'Neill remarked in an interview.
This landmark approval signifies that when patients and families receive the devastating diagnosis of Sanfilippo syndrome, they will now be provided with a glimmer of hope and a course of treatment.
Written by urgent.news from STAT News's reporting — not their text. Machine-written — may contain errors; check the original before relying on it.
