Research programme:microdystrophin gene therapies- Nationwide Childrens Hospital

Drug Profile

Research programme:microdystrophin gene therapies- Nationwide Childrens Hospital

Latest Information Update: 13 Jan 2017

Price : $50

At a glance

  • Originator Nationwide Children's Hospital
  • Class Gene therapies
  • Mechanism of Action Gene transference
  • 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

Highest Development Phases

  • Preclinical Duchenne muscular dystrophy

Most Recent Events

  • 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)
  • 10 Jan 2017 Nationwide Children’s Hospital plans a phase I/II trial for Duchenne muscular dystrophy in USA
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