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February 06, 2025

Ultragenyx to Host Conference Call for Fourth Quarter and Full-Year 2024 Financial Results and Corporate Update

NOVATO, Calif., Feb. 06, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today announced that it will host a conference call at

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February 05, 2025

Ultragenyx Announces New Data Demonstrating that Treatment with UX111 AAV Gene Therapy Significantly Improved Clinical Function Across Multiple Developmental Domains in Children with Sanfilippo Syndrome Type A (MPS IIIA) Correlated with Sustained Reductions in CSF-HS

Modified intent-to-treat group demonstrated +22.7 point (p0.0001) treatment effect in the mean Bayley-III cognitive raw score compared to natural history data (ages 24-60 months) Older children with more advanced disease demonstrated retention of clinically meaningful functional abilities,

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