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We have one of the largest and most diverse pipelines in rare disease.
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View more news >Ultragenyx Announces First Patient Dosed in Pivotal Phase 3 Aspire Study Evaluating GTX-102 in Angelman Syndrome
Ultragenyx Submits Biologics License Application to the U.S. FDA for UX111 AAV Gene Therapy for the Treatment of Sanfilippo Syndrome Type A (MPS IIIA)
Rare perspective
Insights and information on our approach to collaborating with rare disease communities, supporting our teammates at Ultragenyx, and developing new therapies for rare disease
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