Hope is within Reach, but we need your help!
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.
Hope for a Cure
Witnessing a child lose abilities and independence is an indescribable pain. Currently, there are no medicines to control it, to slow it down, or to heal it. Parents are trapped in a hopeless inability to help their children, and there has been no viable prospect of finding treatment or a cure. Until NOW.
Finding Hope for FRRS1L had been working the last 2 years with a team of top gene therapists at the University of Texas Southwestern (UTSW). The team had the challenge of breeding mice with FRRS1L disease, and then developing a gene replacement treatment. FRRS1L bred mice are weak, fragile, and struggle to move and function, just like our FRRS1L children. However, our research proves that when treated with gene replacement therapy the mice come back to function. We want that for all current and future FRRS1L children! We can do it with your help! We can change the world with a cure!
Every day is a fight for these kids, but you can help them.
Help us fund treatment!
