Drug Profile

CLN8 gene therapy - Amicus Therapeutics

Latest Information Update: 28 Oct 2022

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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

28 Oct 2022 No recent reports of development identified for preclinical development in Neuronal ceroid lipofuscinosis in USA
20 Sep 2018 Celenex has been acquired and merged into Amicus Therapeutics
20 Sep 2018 Preclinical trials in Neuronal ceroid lipofuscinosis in USA (unspecified route) prior to September 2018 (Amicus Therapeutics pipeline)

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