Frizzle Treatment Development
Treatment Development Goal= Fall 2026
Finding Hope for FRRS1L is developing gene replacement therapy for Frizzle (FRRS1L) disease. The goal is to dose the first Frizzle patients by fall of 2026. Although we have a plan with our partners to develop treatment, we still need to raise the needed funds to cover the clinical trial and required natural history study. Be a part of this amazing mission to get life saving treatment developed for Frizzle. Donate today!
Treatment Development Process
The organization is currently at the stage of preparing an IND and for the launch of a clinical trial, with the goal to begin by fall 2026.
Note: Our projected timeline for treatment development is completely dependent on funds being raised to pay for each stage. We hope to raise the funds needed as soon as possible to begin a clinical trial this fall.
Treatment by Fall 2026 is possible with your support!
Our race to lifesaving treatment has begun. We have a goal to begin treating Frizzle patients by fall of 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.