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729,586 Results for "ultragenyx pharmaceutical inc".
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Rare diseases
Ultragenyx Takes Second Hit as FDA Rejects Gene Therapy for Sanfilippo Syndrome
The FDA cited manufacturing issues but did not flag problems with Ultragenyx’s data package for UX111, with the biotech noting that the regulator found its neurodevelopmental findings for the gene therapy to be “robust.”
July 14, 2025
·
2 min read
·
Tristan Manalac
Press Releases
Ultragenyx to Participate at Investor Conferences in September 2025
September 1, 2025
·
1 min read
Rare diseases
Ultragenyx, Mereo Plummet After Late-Stage Trial for Rare Genetic Disorder Appears To Fall Short
Partners Ultragenyx and Mereo BioPharma saw their stocks drop by 21% and 30%, respectively, after announcing that the Phase II/III study of their osteogenesis imperfecta candidate will proceed to final analysis, implying it did not show sufficiently strong results at an interim analysis.
July 10, 2025
·
2 min read
·
Tristan Manalac
Press Releases
Ultragenyx Reports Second Quarter 2025 Financial Results and Corporate Update
August 6, 2025
·
14 min read
Opinion
Angelman Syndrome Race Heats Up Among Ionis, Ultragenyx and Neuren
Ionis and Ultragenyx are competing to develop oligonucleotide treatments for Angelman syndrome, but will Neuren’s peptide catch up?
March 17, 2025
·
3 min read
·
Jia Jie Chen
Press Releases
Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) - August 21, 2025
August 22, 2025
·
1 min read
Press Releases
Ultragenyx to Host Conference Call for Second Quarter 2025 Financial Results and Corporate Update
July 31, 2025
·
1 min read
Press Releases
Ultragenyx Completes Enrollment of Phase 3 Aspire Study Evaluating GTX-102 for the Treatment of Angelman Syndrome
July 31, 2025
·
7 min read
Press Releases
Ultragenyx Announces Positive Longer-term Data from Phase 3 Study of DTX401 AAV Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa)
September 9, 2025
·
11 min read
Press Releases
Ultragenyx Receives Breakthrough Therapy Designation for GTX-102 in Angelman Syndrome
June 27, 2025
·
7 min read
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