Drug Profile

Research programme: dystrophin replacement therapeutics - Orna Therapeutics

Alternative Names: Duchenne muscular dystrophy therapeutics - Orna Therapeutics

Latest Information Update: 24 Jun 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

16 May 2022 Early research in Duchenne muscular dystrophy in USA (Parenteral) as of May 2022 (Orna Therapeutics pipeline, May 2022)
16 May 2022 Pharmacodynamics data from a preclinical study in

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