Clinical trials: Hereditary Inclusion Body Myopathy, open to volunteers
- Condition Hereditary Inclusion Body Myopathy
- Status Open to volunteers
- Funder type Industry (pharma, biotech, devices)
GNE Myopathy
First-in-human Study of UX016 in GNEM
The goal of this study is to assess the safety and dose of UX016 and its impact on muscle strength in adults with GNE Myopathy (GNEM).
- Trial name
- NCT07511556
- Testing
- UX016
- Where
- 2 sites in United States
- Volunteers
- 24 people
- Sponsor
- Ultragenyx Pharmaceutical Inc