Frizzle Treatment Development
Treatment Development Goal= September 2026
Finding Hope for FRRS1L is developing gene replacement therapy for Frizzle (FRRS1L) disease. The goal is to dose the first Frizzle patients by September of 2026. Although we have a plan with our partners to develop treatment, we still need to raise the needed funds to cover the manufacturing of the drug that will be given as a one-time dose to patients, as well as the clinical trial. Be a part of this amazing mission to get life saving treatment developed for Frizzle. Donate today!
Treatment Development Process
The organization is currently in the manufacturing process for treatment development; while also preparing for clinical trials to begin by September 2026.
Treatment by September 2026 is possible with your support!
Our race to lifesaving treatment has begun. We have a goal to begin treating Frizzle patients by Sept. 2026, but it will only happen with your support! Help us fund the cure! Donate today to help fund the manufacturing of the drug that will be a one time injection for Frizzle patients!
All donations are tax deductible, and go directly to fund the development of FRRS1L gene therapy treatment. Your gift will help ALL children with FRRS1L. That is older children who have lost hope, younger children who haven’t yet regressed, and unborn children who will inherit this disorder. This research also moves forward the science that may result in cures for other genetic disorders.