RARE Reports New Upbeat Data From Sanfilippo Syndrome Drug Study

Ultragenyx Pharmaceutical (RARE) reported new findings from an ongoing, pivotal study of its investigational candidate, UX111 (ABO-102) AAV gene therapy, for Sanfilippo syndrome type A (MPS IIIA).Per the data readout from the phase I/II/III Transpher A study, treatment with the candidate resulted in a statistically significant improvement in Bayley-III raw scores for cognition, receptive communication and expressive communication in MPS IIIA patients. Compared to natural history data from untreated patients ...