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745,185 Results for "ultragenyx pharmaceutical inc".
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Gene therapy
Ultragenyx’s Gene Therapy Shows Long-Term Efficacy in Two Sanfilippo Studies
The FDA in July last year declined to approve UX111 for Sanfilippo syndrome, a rare neurodegenerative disorder, citing manufacturing issues. Ultragenyx Pharmaceutical resubmitted its application ten days ago, expecting a six month review time.
February 9, 2026
·
2 min read
·
Tristan Manalac
Phase III
Ultragenyx Loses $1B in Market Value as Bone Drug Fails To Reduce Fractures
With a pair of Phase III trial flops, Ultragenyx will explore cost reductions as analysts turn attention to an upcoming Angelman syndrome readout.
January 2, 2026
·
2 min read
·
Annalee Armstrong
Press Releases
Ultragenyx to Participate at Investor Conferences in March 2026
February 24, 2026
·
1 min read
Press Releases
Ultragenyx Reports Fourth Quarter and Full Year 2025 Financial Results and Corporate Update
February 12, 2026
·
12 min read
Press Releases
Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) - February 20, 2026
February 22, 2026
·
1 min read
Press Releases
Ultragenyx to Host Conference Call for Fourth Quarter and Full-Year 2025 Financial Results and Corporate Update
February 6, 2026
·
1 min read
Press Releases
Ultragenyx to Present at the 44th Annual J.P. Morgan Healthcare Conference
January 8, 2026
·
1 min read
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 Announces Positive 36-Week Data from Phase 3 Study of DTX301 AAV8 Gene Therapy for the Treatment of Ornithine Transcarbamylase (OTC) Deficiency
March 13, 2026
·
9 min read
Press Releases
Ultragenyx Provides Financial and Business Updates at J.P. Morgan Annual Healthcare Conference
January 12, 2026
·
7 min read
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