Drug Profile

Research programme: gene therapies - Ultragenyx Pharmaceutical

Alternative Names: AAV-based gene therapies - Ultragenyx Pharmaceutical

Latest Information Update: 28 Apr 2024

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At a glance

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity No

No development reported Early infantile epileptic encephalopathy 2; Metabolic disorders

28 Apr 2024 No recent reports of development identified for research development in Metabolic-disorders in USA (Parenteral)
28 Nov 2022 No recent reports of development identified for research development in Early-infantile-epileptic-encephalopathy-2 in USA (Parenteral)
31 Mar 2020 REGENXBIO and Ultragenyxto develop gene therapy for Rare metabolic diseases

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