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- Ultragenyx—Treatment of Rare and Ultrarare Genetic Diseases
Ultragenyx is focused on developing first-ever approved treatments for rare and ultrarare diseases and moving the rare disease community forward
- pipeline - Ultragenyx
Ultragenyx has a diverse pipeline of clinical development programs for rare and ultrarare genetic diseases
- Medicines - Ultragenyx
Ultragenyx has developed first-ever treatments for multiple rare diseases at a speed faster than the industry average
- Science - Ultragenyx
The Ultragenyx approach to developing new therapies for rare and ultrarare genetic disease is grounded in matching the best treatment modality with a specific disease
- Newsroom—Ultragenyx
Access news releases, presentations, and blog posts to follow the latest developments at Ultragenyx
- Research - Ultragenyx
Ultragenyx focuses its scientific research on developing life-changing therapies for patients with serious rare and ultrarare genetic diseases
- Ultragenyx Announces FDA Acceptance and Priority Review of the . . .
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 For more information on Ultragenyx, please visit the company's website at: www ultragenyx com
- About Our Gene Therapy Manufacturing Facility—Ultragenyx
Ultragenyx has a robust clinical-stage gene therapy pipeline, with three candidates in pivotal studies and others in earlier-stage development At the core of our proprietary gene therapy platform is our clonal Producer Cell Line (PCL) technology
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