A Path To A Cure

Gene Therapy Basics

Developing gene therapy takes years of partnerships, work, and millions of dollars. We are grateful to have a community of families invested in bringing awareness to NARS1 disease as well as Dr Efthymiou researching the gene so diligently. That alone won't be enough to cure this devastating disease! We aim to raise $4 million to reach this goal.

Our next agenda item in our "Path to Cure" will be to fund a natural history study so we can better understand how this disease presents and progresses in individuals. Natural history studies help to frame the disease and give better guidance to drug development from a population standpoint. We need your help to start this process!!