2025 is focused on the next steps in treatment development for Frizzle patients, which includes toxicolgogy testing and manufacturing. In 2024 we raised the funds needed to move forward with pursing toxicolgy testing, which is required by the US FDA. As we conduct testing we are looking ahead to funding the manufacturing of the actual drug that will treat Frizzle patients. Frizzle families and patients are excited and hopeful to get treatment developed that will bring their children back to function.

There is now Hope for TREATMENT!

To begin treating patients in 2026/2027 we need to fundraise the remaining amount of funds to complete the treatment development process. Join us on this amazing journey to change lives and change the world by donating today!

We need your help to develop lifesaving treatment!

HELP FUND A CURE

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HELP FUND A CURE 〰️ Click to Donate 〰️

FUNDRAISING UPDATE

  • Goal 1: COMPLETED

  • GOAL 2: COMPLETED

  • GOAL 3: (Current Stage)

    • Fundraising Goal: $1 million by Dec. 31, 2024 - ON TRACK FOR COMPLETION

    • Toxicology Testing: This step in treatment development is required by the US FDA. We can now begin this process with the funds raised.

  • Goal 4: (Summer 2025)

    • Fundraising Goal: $3 million to fund the manufacturing of the drug/treatment

  • Goal 5: (Spring 2026)

    • $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.

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, results are in from FRRS1L mouse models and gene replacement treatment worked to bring function back to FRRS1L diseased mice! This is life changing news, that requires more action and funding. Would you join us in making treatment a reality for sick FRRS1L children?

Donate today to help us fund treatment development for FRRS1L children!

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!

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 a Cure and how you can help!

 

Donate to Help Fund a Cure for FRRS1L

Help provide life saving treatment to children with FRRS1L. All donations are tax deductible, and go directly to fund the research and 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.

It wasn't always like this for children with FRRS1L gene disorder.

Before the disorder took full effect, parents knew and saw their children to be busy toddlers. FRRS1L is a critical brain encoding gene that affects all cells in the brain. Children with this gene disorder develop, albeit delayed, until age two when they begin having seizural activity that causes regression and loss of function.

Learn more about FRRS1L

Within months, or even weeks, children lose their ability to move, speak, feed orally, and control their head and their body.

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.

Learn more about our Hope for a Cure.

REGISTER your FRRS1L Child

By registering your child/children with Finding Hope for FRRS1L, it provides valuable information that will help us better understand and track the global map of the FRRS1L community; it will inform researchers conducting a natural history study of FRRS1L; and it also allows us to keep you informed through email updates and news on the progress towards developing gene replacement therapy. Registering your child is completely optional.

Finding Hope for FRRS1L Mission

  • FIND a Community

    Create a community of Hope for FRRS1L gene disorder children and families to connect, communicate, collaborate, and support one another.

  • HOPE for a Cure

    Serve to fund research and development to find a cure for FRRS1L gene disorder and other treatments that would improve the quality of life of children with FRRS1L.

  • FRRS1L awareness

    Share information and resources with the public, medical professionals, and researchers to increase the awareness and understanding of FRRS1L.

Our Community

  • Emilio

  • McKayla

  • Arturo

Goals

Families

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As we fundraise and word gets out, there are more families contacting us that have children with FRRS1L genetic disorder. Our goal is to get them connected with providing: key information, a link to join the FRRS1L Support group on Facebook, access to current research efforts, and the ability to join our efforts in funding a cure.

Fundraising

Raise money to fund the development of gene replacement therapy treatment for FRRS1L genetic disorder children. Help us fund a cure!

Medical Professionals

Reaching out to doctors, medical professionals, and organizations to increase awareness of FRRS1L.