Led by Kevin Flanigan, MD at Nationwide Children’s Hospital, this investigator initiated therapy will focus on genetic and molecular characterization of inherited neuromuscular diseases and toward the development of therapies directed toward these diseases.
Research focus: Exon skipping (Duplication).
Status: Pre-clinical. Submitting pre-IND package to FDA.
Funding Need: $500,000 – In collaboration with Duchenne community partners in initial funding. Total of $1.4M including scale-up of a gene-therapy delivery vector needed for a clinical trial.
“CureDuchenne recognized early on the importance of our research focused on rare and duplication mutations and we wouldn’t be here today without their support. CureDuchenne supported the development of the mouse model and the personnel to work on this research. CureDuchenne plays a vital role in funding early phase research projects to help accelerate their development.” — Dr. Kevin Flanigan