Latest Information Update: 20 Nov 2015
At a glance
- Originator Nationwide Children's Hospital
- Developer Milo Biotechnology
- Class Gene therapies
- Mechanism of Action Activin inhibitors; Follistatin stimulants; Gene transference; Myostatin inhibitors
Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
On Fast track
Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.
- New Molecular Entity No
Highest Development Phases
- Preclinical Muscular atrophy
Most Recent Events
- 20 Nov 2015 Preclinical trials in Muscular atrophy in USA (unspecified route) before November 2015