HAPPY NEW YEAR

Help make THIS YEAR the year for FRRS1L TREATMENT!!

Our goal is to begin treating Frizzle patients by September of this year with the one-time dose of gene replacement therapy. We have a plan in place, partners ready, and a schedule to get this drug developed, but we just need the funds to get it to the finish line and in patients. We need to raise $4 million to pay for both the manufacturing of the actual drug that will be given to patients, as well as the costs of treating patients in a clinical trial. Every donation will go directly to changing lives and changing the world! Let’s stop Frizzle disease together!

A young girl in a hospital bed with a nasal oxygen tube, surrounded by two women holding her hands and talking to her. The girl has a bee-themed neck pillow. There is medical equipment and monitors on the wall.

Treatment by September 2026 is possible with your support!

Our race to lifesaving treatment has begun. We have a goal to begin treating Frizzle patients by Sept. 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.

DONATE HERE

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!

A man is giving a presentation in a conference room, standing in front of a large screen displaying a scientific study about gene replacement in mice. Several people are seated at tables, listening, with some taking notes or working on laptops.
A female scientist wearing a white lab coat looking through a microscope in a laboratory with shelves of supplies and equipment.
A classroom or conference room with two women sitting at a table, watching a large screen displaying a close-up of a mouse on a white surface. There is a woman standing at a podium near a computer in the background.
A graphic infographic titled 'Ending Hope' with a pathway and flags marking milestones. The pathway starts with 'Pre-Clinical Work Completion' at $400,000, then 'Toxicology' at $1 million, 'Manufacturing' at $3 million, and 'Clinical Trials' at $2 million. Checkmarks mark each milestone.

FUNDRAISING UPDATE

Current Total Program Funding Need: $4 million

  • Goal 1: COMPLETED

  • GOAL 2: COMPLETED

  • GOAL 3: COMPLETED

    • Fundraising Goal: $1 million by Dec. 31, 2024

    • 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: (Current Stage)

    • 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: (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.

Fighting For Two Lives

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Fighting For Two Lives ~

Viviana found herself fighting for two lives. For her son Arturo who has Frizzle (FRRS1L) a devastating genetic disease, and her own life as she was diagnosed with Breast Cancer. Watch her story of strength and determination. She is thankful there is lifesaving treatment for breast cancer, which saved her life; now she fights even harder to get lifesaving treatment funded for Frizzle to save her son. Finding Hope for FRRS1L has discovered lifesaving treatment for Frizzle, the organization has a plan to develop it, and a goal to dose patients starting in Sep. 2026, but the only roadblock standing in their way is funding.

Help fund lifesaving treatment for Frizzle by donating today!

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!

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.

REGISTER TODAY
Documentary Trailer and Info Here

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!

Finding Hope for FRRS1L Mission

  • Four figures connected to a heart in the center, symbolizing love or connection.

    FIND a Community

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

  • Abstract black and white design with a heart at the center and swirling lines around it.

    HOPE for a Cure

    Serve to fund research and development of gene replacement therapy treatment for FRRS1L genetic disease.

  • Outline of a human head with a heart shape inside it

    FRRS1L awareness

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

Learn About Us

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!

Donate Today

Medical Professionals

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