2026 the Year for Frizzle TREATMENT
Donate today to help us get this treatment to stuggling Frizzle patients this year!
Our goal is to begin treating Frizzle patients by September of this year with a one-time gene replacement therapy.
We have a clear plan, committed partners, and a defined timeline. What remains is raising the funds needed to bring this treatment across the finish line and into patients.
We must raise $4 million to cover both the manufacturing of the drug and the costs of treating patients in a clinical trial.
Every donation goes directly toward changing lives and changing the world! Together, we can stop Frizzle disease!
QUICK LINKS
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DONORS and PARTNERS
We are looking for donor partners and champions to help us get this treatment to the finish line and in patients by September this year! Learn more about our treatment development timeline, fundraising plan, and ways to give here.
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PHYSICIANS and RESEARCHERS
Researchers, doctors, and medical professionals looking for more information about Frizzle disease, published research, and treatment information, learn more here.
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FRIZZLE (FRRS1L) FAMILIES
Are you a parent or caregiver of a Frizzle (FRRS1L) patient? Learn more here. Learn more about our special community and register your Frizzle child or children today here!
FUNDRAISING UPDATE
Current Funding Need: $4 million
Goal 1: COMPLETED
GOAL 2: COMPLETED
GOAL 3: COMPLETED
Goal 4:
Fundraising Goal: $3 million to fund the manufacturing of the drug/treatment
Current Status of Goal: $1 million raised of $3 million goal
Goal 5:
$2 million for Clinical Trials to treat Frizzle children
Note: Our projected timeline for fundraising and 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 keep the treatment development process moving; however, it is subject to change.
Treatment by September 2026 is possible with your support!
Our race to treatment has begun. We have a goal to begin treating Frizzle patients by Sept. 2026, but it will only happen with your support! 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.
Pre-clinical Data shows Restoration of Function
Our pre-clinical research on FRRS1L mouse models showed the recovery of function through gene replacement therapy. The gene replacement therapy given to FRRS1L diseased mice worked to bring function back to FRRS1L diseased mice brains and bodies! This is life changing news! We want recover for Frizzle patients and are working to get treatment developed this year. Would you join us in making treatment a reality for sick Frizzle patients this year?
Donate today to help us fund treatment development for Frizzle patients!
Within weeks children lose their ability to move, speak, eat, and control their body.
We see these children looking at a toy they once held. We know they want to play with it, but they physically can’t. They are trapped.
Learn about their Hope for treatment and how you can help!
Watch the trailer and learn more about our One Dose Documentary. Follow along on our journey to develop lifesaving treatment for Frizzle patients around the world!