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We’re Funding Sight-Saving Clinical Trials to End Childhood Blindness.

We’re on the cusp of funding a clinical trial for a revolutionary new gene therapy. Our goal is to cure retinitis pigmentosa caused by Bardet-Biedl Syndrome (BBS) – a rare but devastating cause of childhood blindness.

There is hope for childhood blindness
funding the best in retinitis pigmentosa research
impacting clinical trials

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You can make an impact on saving childhood eyesight by entering our giveaway fundraisers.

See our Feature on Good Morning America!

Luke’s story of seeing the world before losing more vision was featured on GMA, helping propel our mission to the national level.

We were told there was nothing we could do about it. Now, we have every reason to hope, if we stand together.

READ OUR STORY

How We’re Going To Beat Childhood Blindness

What is Retinitis Pigmentosa (RP)?

Retinitis pigmentosa (RP) can be caused by many conditions. Luke’s condition is caused by Bardet-Biedl Syndrome (BBS), which affects multiple organ systems, including the eyes. There is a slow, progressive loss of vision until many BBS patients become blind in their teenage years.

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We have a chance to change the future for children like Luke.
We were told there was no cure.

Now We're Funding Clinical Trials

The medical research involved in vision saving treatment and restorative therapy is advancing rapidly. This is excellent news! However, funding is significantly limited. Private funding, such as the money we raise, remains the main driver for much of this innovation. Your support is vital to saving children’s vision.

    We Have the Best Helping

    Axovia Therapeutics is working on Luke’s exact condition.

    FINDING THE BEST IN RESEARCH

    They had progressed to the point of having completed animal studies in preparation for human clinical trials. At this point they need funds to advance to these clinical trials. It is uncommon for rare disease research to get government funding or funding from private pharmaceutical companies. Thus, the burden often falls on non-profits and philanthropists to fill in the gap between research and a cure. This is the difficult task we have undertaken.

    We’re Funding a Clinical trial between Axovia therapeutics and Mayo Clinic.

    WHERE OUR FUNDS GO

    We’ve set up our non-profit to minimize expenses and create a pipeline directly to Axovia Therpauetics.  They are only team in the world developing a therapy for retinitis pigmentosa due to BBS-1, and the planned clinical trial will take place at Mayo Clinic in Rochester. A similar gene therapy, Luxturna, is FDA approved to treat retinitis pigmentosa due to Leber congenital amaurosis. Axovia’s treatment uses a similar gene therapy model with some minor changes. With each successive gene therapy treatment that is successful, it becomes easier to develop treatments for more genetic subtypes and thus treat a wider patient population.

    Elevating the Rare Disease Community

    OUR PRESENT AND FUTURE

    With your help, we can streamline the FDA approval process for rare diseases, which is incredibly time-consuming and expensive. Axovia is targeting BBS-1 today, but our success will elevate the entire community in the near future. It’s very important to have these individual wins because, without a proven model, we wouldn’t be where we’re at right now. This treatment is ready to go, and the next step we’re funding is human trials.

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    How You Can Support

    DONATE

    Every donation, no matter how large or small, makes an impact. The efficiency of our mission to support clinical research makes every dollar count.

    DONATE NOW

    GIVEAWAYS

    Donate and help fund life-saving research for children. Your donation enters you into giveaways like the 2023 Beast Mode Adventure Van.

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    VOLUNTEER

    Create your own events or help out at one of ours! Be a part of our operations, write letters to donors, or help solicit donations: the possibilities are endless.

    CONTACT US
    there is hope

    Meet the Team

    We founded the nonprofit when we found out our son has a rare genetic condition and is slowly going blind. But that really only scratches the surface in terms of our motivation. Since learning of Luke’s diagnosis , we have become strongly connected to the rare-diseases community. While rare diseases are actually not unusual, with more than 7,000 diseases classified by the FDA as rare, each one individually often affects only a small number of people. Thus many rare diseases don’t receive enough attention and resources to help fund research for adequate treatments or even possible cures.