Ultragenyx Announces Completion of Dosing Across Stage 1 Cohorts in Pivotal Phase 1/2/3 Cyprus2+ Study Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease
Safety and initial efficacy data from Stage 1 expected in the first half of 2024
Dose selection and initiation of Stage 2 to follow in the second half of 2024
Related news for (RARE)
- Ultragenyx Announces Data Demonstrating Treatment with UX111 Results in Significant Reduction in Heparan Sulfate Exposure in Cerebrospinal Fluid Correlated with Improved Long-term Cognitive Function in Patients with Sanfilippo Syndrome Type A (MPS IIIA)
- Ultragenyx Receives PRIME Designation from European Medicines Agency (EMA) for GTX-102 for the Treatment of Angelman Syndrome
- Ultragenyx anuncia la aprobación de Dojolvi® (triheptanoína) para su inclusión en el Compendio Nacional de Insumos para la Salud de México en el tratamiento de los trastornos de oxidación de ácidos grasos de cadena larga (LC-FAOD)
- Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)