Drug Profile

Research programme: CNS oligonucleotide therapeutics - BioMarin Pharmaceutical

Latest Information Update: 02 May 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 Yes

23 Apr 2024 Preclinical trials in CNS disorders in USA (Parenteral) before April 2024 (BioMarin Pharmaceutical pipeline, April 2024)

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