Hello everyone,

We’re Kris and Grant, the very proud parents of our beautiful daughter, Rylee.

Although Rylee has faced more challenges in her short life than most people experience in a lifetime, she continues to greet the world with the brightest, most heartwarming smile. Her resilience inspires us every single day.

Rylee lives with an ultra-rare neurodevelopmental genetic disorder FBXO11. This condition affects how her brain processes and clears proteins, impacting nearly every aspect of her development and daily functioning. Because FBXO11 is so rare, there is very little information about what the future holds or what milestones she may or may not achieve. When she was diagnosed, there were only 84 known cases worldwide.

Rylee entered the world with a bang.

We had one beautiful day at home before everything changed. What we thought would be a simple overnight hospital stay for jaundice quickly became a nightmare. Rylee repeatedly stopped breathing, and doctors had to perform life-saving resuscitations. We came terrifyingly close to losing her four times. We are forever grateful that she was already in the hospital when those emergencies happened.

Because of those breathing difficulties, Rylee has required oxygen every time she sleeps since she was a newborn.

We spent the next six weeks in the hospital while doctors performed countless tests in search of answers. Finally bringing her home was an incredible relief, but the challenges didn’t stop there.

Despite around-the-clock care, Rylee struggled to gain weight. At five months old she was still failure to thrive, was admitted back to the hospital, and underwent surgery to have a G-tube placed. Today she relies on her feeding tube for hydration and nutrition.

When we first learned about her diagnosis, we held onto hope that because so little was known about FBXO11, perhaps the predicted challenges wouldn’t all become reality.

Unfortunately, the opposite has happened.

Rather than catching up, Rylee has fallen further behind developmentally and has even lost some abilities she once had. She was born with a strong ability to feed orally but has since become almost completely dependent on her feeding tube. Despite working hard in multiple therapies regularly, she is delayed in every developmental milestone—except for one.

Her smile.

It is impossible to put into words the joy that smile brings to everyone who meets her.

Every week, Rylee works incredibly hard through physiotherapy, occupational therapy, and speech therapy to build strength, coordination, and communication skills. No one can tell us what her future will look like, but she never stops trying. Watching her determination has taught us more about courage than we ever thought possible.

As her parents, we believe it’s our turn to fight just as hard for her.

We have already committed a significant amount of our own resources to begin researching and confirming the possibility of developing a safe gene therapy for FBXO11. While this process is complex, lengthy, and incredibly expensive, it represents the greatest hope we have of improving Rylee’s quality of life.

After finally emerging from survival mode, we’ve spent countless hours researching, speaking with specialists, scientists, and medical professionals to understand what might be possible. The first five years of life are crucial for brain development so time is crucial to give her the best chance for the most impactful change.

We will love and care for Rylee for the rest of our lives without hesitation.

But like every parent of a child with complex medical needs and special needs, we carry a heavy fear that is impossible to ignore: what happens when we’re no longer here?

Our greatest dream isn’t perfection. It’s that one day Rylee will be able to communicate her needs, understand the world around her, make her own choices, and stand up for herself. We want her to have every opportunity to live with as much independence, dignity, and joy as possible.

Every parent would move mountains for their child.

This is ours.

If you’re able to support Rylee—whether by donating or simply sharing her story—you are giving our family hope. Hope that one day this research could change not only Rylee’s future, but potentially the future of other children diagnosed with this rare disorder.

From the bottom of our hearts, thank you for taking the time to read our story and for being part of Rylee’s journey.

With love and gratitude,

Kris, Grant, Rylee & big sister Addy

Please follow @ryleesroad for frequent updates about her day to day journey. See below for more information about Gene Therapy

What is Gene therapy and how does it work?

Gene therapy is a medical technique that treats or prevents disease by modifying, repairing, or replacing a person's genetic material (DNA or RNA). Instead of using traditional drugs or surgery, it fixes the underlying genetic errors responsible for health conditions at the cellular level.

Our genes provide the blueprints (instructions) for making proteins, which run our bodies. When a gene has a mutation (an error in its code), it may produce a faulty protein or no protein at all, leading to disease. Gene therapy works by delivering new instructions to the cells through three main strategies,

Why Gene Therapy for Rylee?

Rylee’s condition is so rare there is very little information about it globally. There’s no treatment or cure. After meeting with medical professionals and research specialists this option has been identified as the only way to help Rylee and improve her expectancy and quality of life.

Why we’re asking for help?

Gene Therapy has advanced greatly in recent years and is a great tool in correcting gene issues, saving lives and improving devastating life prognosis for people affected. In the medical field there isn’t funding or resources available to research rare disease's/conditions such as Rylee’s. To safely research, test and create a Gene Therapy is incredibly expensive. Quotes we’ve received are 2-4.5 million dollars which is a substantial amount of money. Unfortunately we haven’t won the lottery and this work isn’t able to be done on a lifelong payment plan. So we’re hoping we can raise awareness and support for her to create and receive this treatment. Which will also open the door for the other families and kids that are affected with this disease in the process.

Fiscal Breakdown of work to be done (approximations given from Medical Institution and Scientific labs)

1. Proof of concept 500-600 thousand

2. Toxicology 1 million

3. Regulatory Filing 250 thousand

4. Manufacturing Clinical Grade 2-3 million

5. Treatment Hospital Administration cost 250 thousand