Biotechnology California, USA-based rare disease company Ultragenyx Pharmaceutical yesterday released new data demonstrating treatment with UX111 (ABO-102) AAV gene therapy led to a statistically-significant improvement in the Bayley-III raw scores for the subdomains of cognition, receptive communication and expressive communication in patients with Sanfilippo syndrome type A (MPS IIIA) compared to natural history data from untreated patients. 6 February 2025