Drug Profile

Research programme: gene therapies - Evozyne/Takeda

Latest Information Update: 28 Feb 2025

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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 Inborn error metabolic disorders; Lysosomal storage diseases

28 Feb 2025 No recent reports of development identified for research development in Inborn-error-metabolic-disorders in Japan (Parenteral)
28 Feb 2025 No recent reports of development identified for research development in Inborn-error-metabolic-disorders in USA (Parenteral)
28 Feb 2025 No recent reports of development identified for research development in Lysosomal storage diseases in Japan (Parenteral)

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