Many researchers are exploring many different avenues in an effort to better understand, treat, or cure SMA. Getty Owl Foundation encourages and supports efforts to attack SMA from many different angles. We also believe in investing our donor’s money wisely by contributing more heavily to those projects bearing the greatest potential.
Dr. Brian K. Kaspar, researcher at Nationwide Children’s Hospital /The Ohio State University, is working on a Gene Therapy Program. The goal of the Program is to deliver specific kinds of genes into the body where they are needed and can be used to halt SMA.
This revolutionary research has the potential to cure Spinal Muscular Atrophy, as well as various other neurological diseases. The pre-clinical data from this project is amazing and shows great potential. No other research project has achieved the results that have been accomplished so far in this Gene Therapy Program.
Based on initial non-binding talks with the Food and Drug Administration there is a realistic timeline of what is necessary to ensure this program reaches human clinical trials.
The Gene Therapy Program needs roughly $1,000,000 to move forward with clinical trials. Getty Owl Foundation proudly supports Dr. Kaspar’s Gene Therapy Program.