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Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.



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April 15, 2024

Ultragenyx Announces Positive Interim Phase 1/2 Data in Patients with Angelman Syndrome After Treatment with GTX-102

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April 12, 2024

Ultragenyx Announces Update to Conference Call Timing to Discuss New Data from GTX-102 in Patients with Angelman Syndrome

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April 12, 2024

Ultragenyx Announces Upcoming Data Presentations at American Academy of Neurology (AAN) 2024 Annual Meeting

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