|Rare Disease Day Activities on the Ivory Coast |
1. The Multi-center Study of ManNAc for GNE Myopathy (MAGINE) is estimated to start on February 28, 2022. This study will last for approximately two years and will recruit fifty-one GNEM patients. Link for more details.
4. Research continues by Dr. S. Mitrani-Rosenbaum on the "Status of GNEM Mouse Model, "Pre-Clinical Assessment of AAVrh74MCK GNE Viral Vector, Therapeutic Potential Robust Activity Despite Lack of Consistent Animal Model for GNE Myopathy." Link for this article.
5. ProDGNE plans to develop a "substrate replacement" to treat GNE Myopathy. This project is in the early clinical stage. Link for more on this research.
Links to GNEM Learning Resources: