We’re proud of the work we’ve been able to accomplish and thankful for the generosity of our supporters as we move toward a stronger future.
Research Milestones
AFBS has made significant milestones toward our mission of finding treatments for NM:
- Developed Standard of Care Protocol for NM + a Guide for families caring for NM
- Funded the first CRISPR/Cas9 Gene Editing project of an NM gene (NEB)
- Developed accurate zebrafish and mouse models for Nebulin and ACTA1 genes
- Developed induced pluripotent stem cell lines from NM patients
Meet Our Community
The Nemaline Myopathy community is dynamic, representing all ages and degrees of severity. Life with NM can vary greatly from one person to another and we want to celebrate and highlight the individuality of our community. You can meet our community members through these special “community spotlights”.
