RARE Submits BLA to the FDA for Sanfilippo Syndrome Gene Therapy
Ultragenyx Pharmaceutical RARE announced that it has submitted a biologics license application (BLA) to the FDA for its UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome...
Ultragenyx Pharmaceutical Inc. Doses First Patient in Pivotal Phase 3 Aspire Study for GTX-102 in Angelman Syndrome
Ultragenyx initiates Phase 3 study of GTX-102 for Angelman syndrome, assessing safety and efficacy in 120 children.Quiver AI SummaryUltragenyx Pharmaceutical Inc. announced the dosing of the first patient...
Insider Sale: President & CEO of $RARE (RARE) Sells 8,273 Shares
EMIL D KAKKIS, the President & CEO of $RARE ($RARE), sold 8,273 shares of the company on 12-10-2024. We received data on the trade from a recent SEC filing. This was a sale of approximately 0.4% of their...