Brief: Ultragenyx: Long-Term UX111 Data Shows Sustained CSF-HS Reductions, Multi-Domain Gains In Children With Sanfilippo Syndrome (Mps Iiia)
03/02/2026 13:09
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 Source text: ID:nGNX5NdJ2w Further company coverage: RARE.O ((Reuters.Briefs@thomsonreuters.com;))
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