Clinical trials: GNE Myopathy, active
GNE Myopathy
Multi-Center Study of ManNAc for GNE Myopathy
GNE myopathy is a rare genetic muscle disease characterized by progressive muscle atrophy and weakness. The disease is caused by mutations in the gene that encodes the enzyme that initiates and regulates N-acetylneuraminic acid…
- Trial name
- MAGiNE (NCT04231266)
- Testing
- ManNAc
- Where
- 10 sites in United States
- Volunteers
- 54 people
- Sponsor
- Leadiant Biosciences, Inc.
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