Ultragenyx: Long-Term UX111 Data Shows Sustained CSF-HS Reductions, Multi-Domain Gains In Children With Sanfilippo Syndrome (Mps Iiia)

Feb 3 (Reuters) - Ultragenyx Pharmaceutical Inc RARE.O:

• ULTRAGENYX ANNOUNCES POSITIVE LONGER-TERM DATA DEMONSTRATING TREATMENT WITH UX111 GENE THERAPY RESULTS IN SUSTAINED, SIGNIFICANT REDUCTIONS IN CSF-HS AND CONTINUED MEANINGFUL IMPROVEMENTS IN CLINICAL FUNCTION ACROSS MULTIPLE DEVELOPMENTAL DOMAINS IN CHILDREN WITH SANFILIPPO SYNDROME (MPS IIIA)

• ULTRAGENYX PHARMACEUTICAL INC - BLA RESUBMITTED TO FDA, SIX-MONTH REVIEW EXPECTED

• ULTRAGENYX PHARMACEUTICAL INC - RESULTS SHOW SUBSTANTIAL BIOMARKER IMPROVEMENTS AND FUNCTIONAL BENEFITS

• ULTRAGENYX PHARMACEUTICAL INC - UX111 WELL TOLERATED ACROSS ALL DOSES