HOPE is within REACH

We have proven in the lab that treatment in FRRS1L mice brings them back to function; now, only with your help, can we fund treatment to help do the same for our FRRS1L children. Learn more about our fundraising plan to get FRRS1L children treatment.

Fundraising Road Map

We started with only a HOPE that needed to be tested in a lab. We had family and friends help us fund the first $400k needed to do the lab work to test FRRS1L mice with gene therapy treatment. We have lab research results showing FRRS1L mice treated with gene therapy treatment COME BACK TO FUNCTION! This is amazing and life changing results! Although the HARDEST WORK in the lab has been done… we have the most expensive, but also MOST IMPORTANT steps ahead to FUND TREATMENT! Our total fundraising goal was $6.5 million, but thankfully we only have $4 million left to raise. Every minute without treatment is a threat to the lives of FRRS1L children. Every donation keeps us racing ahead to the ultimate goal of life changing treatment.

Change THEIR world, change OUR world, change THE WORLD!

DONATE TODAY!

Current Need: $4 million

A timeline infographic titled 'ENDING HOPE' showing funding stages for medical research. Stages include pre-clinical work at $400k, toxicology at $1 million, manufacturing at $3 million, and clinical trials at $2 million.

FUNDRAISING UPDATE

  • Goal 1: COMPLETED

  • GOAL 2: COMPLETED

  • GOAL 3: COMPLETED

  • Goal 4: (Current Stage)

    • 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.

Be a Part of Changing the World

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    GIVE

    Donate today to help fund a cure. All donations are tax deductible.

    DONATE
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    SHARE

    Share our emails, posts, links, and information to get the word out that we need help funding life changing treatment.

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    FRRS1L AMBASSADOR

    Join us in this life changing cause by becoming an official FRRS1L Ambassador.

    BECOME AN AMBASSADOR
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    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!

    BECOME A CORPORATE CHAMPION

JOIN US IN CHANGING THE WORLD!

JOIN US IN CHANGING THE WORLD!

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We are looking for innovative and visionary corporate donors to be a part of our life saving, world changing, and ground breaking cause. Children from around the world with FRRS1L returning to function will be amazing news. We look forward to celebrating that day sooner than later, but not without the vital help of partners. Join us as an official CORPORATE CHAMPION!

Join Us as a CORPORATE CHAMPION

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 giving a presentation in a conference room with a large screen displaying a slide about a pre-clinical study involving gene replacement in mice. Several people are seated at tables, some with laptops and drinks.
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TAKE ACTION - DONATE TODAY
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A young boy with curly hair sitting in a stroller, wearing a green T-shirt with a yin-yang symbol, and a black cap with an adventure-themed patch, with his mouth slightly open and tongue sticking out.
A young girl with dark hair lying in a hospital bed, connected to medical equipment with a nasal tube. She is wearing a pink sleeveless top with a floral and unicorn pattern and has colorful hair bows with glitter decorations in her hair.

HELP FUND A CURE

These sweet, but struggling children, now have HOPE that treatment is possible, but we need your help to fund it.

DONATE TODAY
A young girl lying in a hospital bed with medical equipment and tubes attached, wearing a green hospital gown with animal patterns, a nasal oxygen tube, and a brace on her right hand.
A young child with curly hair and blue glasses smiling while playing with a cookie on a table, wearing a bib that says 'Mini & Mighty' in a kitchen setting.
A young girl with down syndrome wearing glasses, lying in a hospital bed, holding a Minnie Mouse plush toy, with colorful paper hearts decorated on the wall behind her.
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