THE TRUTH ABOUT: Ultragenyx | History Defined

FDA Clears First Gene Therapy for Sanfilippo Syndrome Type A with Shipments to Treatment Centers Within 60 Days

The FDA approved Fayuvi, the first gene therapy for Sanfilippo syndrome type A. What families should know about access, cost and safety checks.

Medical Daily · Sep 18

A Gene Targeting Therapy for Angelman Syndrome Failed Its Final Trial, Leaving Families Without an Approved Option

Ultragenyx says apazunersen missed both endpoints in the Phase 3 Aspire trial for Angelman syndrome, and no approved therapy exists.

Medical Daily · Sep 7

Gene Therapy for Glycogen Storage Disease Type Ia Wins Accelerated Approval Priced at 2.7 Million Dollars

FDA granted accelerated approval to Genglycos for glycogen storage disease type Ia. It reduces cornstarch use, and the long-term benefit is unproven.

Medical Daily · Aug 21

1 Biotech Stock to Buy for 104% Upside Potential

Ultragenyx Pharmaceuticals is a beaten-down biotech stock trading at a steep discount to consensus price target estimates.

Barchart · Jun 13, 2024

Family of Henrietta Lacks files new lawsuit over cells harvested without her consent

The family of Henrietta Lacks has filed another lawsuit accusing a second company of unjustly profiting off her cells for generations

The Independent UK · Aug 10, 2023