Drug Profile

Research programme: microdystrophin gene therapies - Nationwide Children's Hospital

Latest Information Update: 28 Feb 2021

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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 Feb 2021 No recent reports of development identified for preclinical development in Duchenne muscular dystrophy in USA
10 Jan 2017 Nationwide Children’s Hospital and Sarepta Therapeutics enter research and option agreement for Microdystrophin gene therapy for Duchenne muscular dystrophy
10 Jan 2017 Preclinical trials in Duchenne muscular dystrophy in USA (unspecified route)

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