Hope is within Reach, but we need your help!
Data is in and it shows treatment is possible for FRRS1L children,
but it won’t happen without your help!
After 2 years of work in the lab, results are in from FRRS1L mouse models and we can prove gene replacement treatment worked to bring function back to FRRS1L diseased mice! This is life changing news, treatment is possible for FRRS1L children, but it requires more action and funding. Would you join us in making treatment a reality for sick FRRS1L children?
Donate today to help us continue the process of producing treatment for FRRS1L children!
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!
Be a Part of Changing the World
-
GIVE
Donate today to help fund a cure. All donations are tax deductible.
-
SHARE
Share our emails, posts, links, and information to get the word out that we need help funding life changing treatment.
-
FRRS1L AMBASSADOR
Join us in this life changing cause by becoming an official FRRS1L Ambassador.
-
CORPORATE CHAMPION
Join us as a Corporate Champion and help change the world. We are looking for innovative and visionary corporate donors to be a part of our life saving, world changing, and ground breaking cause. Join us as an official CORPORATE CHAMPION!
Gene Therapy
Currently we are pursuing the research and development of gene replacement therapy for FRRS1L gene disorder with Dr. Minassian at UT Southwestern. The team has been working with FRRS1L mice and has research to prove that gene replacement therapy works to bring back function in FRRS1L mice. We are at an exciting place with the research and are confident with funding and support, that we will reach clinical trials for FRRS1L children.
To learn more about gene therapy please check out the following resources:
Every day is a fight for these kids, but you can help them.
Help us fund treatment!
