Hailee’s Journey: A Story of Resilience and Hope in the Face of Morquio A Syndrome

At just three years old, Hailee began to experience pain that set her apart from her peers. While other children her age were running around and playing, she often asked to be carried by her parents, Tiffany and Jason. As her mother recalls, “We started to notice there were some things going on developmentally. Physically and neurologically, everything seemed fine, but her elbow shape and hands were growing differently.” Hailee was also shorter than her friends, but given that Tiffany is petite, doctors initially dismissed it as a familial trait.

However, the situation escalated in January 2019 when Hailee developed a fever and refused to walk due to severe neck pain. This alarming change prompted Jason to take her to the Seattle Children’s Emergency Department, where a battery of tests—including blood work, CT scans, ultrasounds, and MRIs—were conducted over several days. “We tried to imagine all the things it could be,” Tiffany recalled, “but the actual diagnosis was far more serious than anything we could have imagined.”

The Diagnosis: Morquio A Syndrome

After four days of uncertainty, the family received heartbreaking news: Hailee had been diagnosed with mucopolysaccharidosis type 4A, commonly known as Morquio A syndrome. This rare genetic disorder is classified as a lysosomal storage disease (LSD), where the body lacks the enzyme necessary to break down sugar chains. As a result, these molecules accumulate in bones, organs, and soft tissues, leading to various health complications.

Morquio A syndrome is one of the rarest forms of LSDs, occurring in about one in every 5,000 to 7,000 births. “How I describe Hailee’s disease to people is that we all have energy and make energy, and our cells go through that process of metabolism,” Jason explained. “In that process, there’s garbage as a byproduct, and most people’s bodies have ‘garbage trucks’—enzymes that take the garbage out. Hailee doesn’t have that, so the garbage builds up and clogs her skeletal systems, causing deformities and an inability to grow.” Approximately one in every 200,000 to 300,000 babies is born with this condition, affecting nearly every part of Hailee’s body except for her brain, and currently, there is no cure.

Navigating Challenges: Financial and Emotional Strain

As if receiving such devastating news wasn’t enough, the family was also grappling with the fact that Jason had recently lost his job. “It was so much life-changing information all at once, and we were terrified,” Tiffany recalled. The financial implications of Hailee’s diagnosis were overwhelming, prompting them to seek assistance from Seattle Children’s Financial Assistance team. They were encouraged to apply for Uncompensated Care, which covers medical expenses, allowing families to focus on their child’s health rather than the costs associated with it.

To their relief, the application process was straightforward, and they were approved for assistance. “In my life, I have experienced financial services at other hospitals in the country, but Seattle Children’s just does it right,” Jason said. “It was the most magical experience anyone can ever have when they’re in need. It was a relief, and I gave the advisor a huge hug.”

Treatment and Ongoing Care

While there is no cure for Morquio A syndrome, Hailee receives enzyme replacement therapy (ERT) through a port in her chest. This treatment acts as a ‘garbage truck’ to help her body eliminate waste. The infusions occur weekly and can last up to seven hours each time, a commitment that Hailee will need for the rest of her life.

Today, Hailee is a vibrant 9-year-old who loves singing, playing Roblox, and dreams of YouTube stardom. Despite undergoing major surgeries—including spinal cord decompression and double hip surgeries—she remains a bubbly and outgoing child. She uses hearing aids and a power wheelchair for mobility, both adorned in sparkly pink, reflecting her joyful personality.

“She’s always trying to make friends,” said her older brother, Jonah. “Wherever we go, she’ll ask people if they want to be her friend because she likes to be friends with everyone.”

Looking Ahead: A Bright Future

With the financial burden eased, Hailee’s family is focused on the positive days ahead. They have joined other families to raise awareness about Morquio syndrome and inspire others to support Seattle Children’s through various initiatives, including community events like the annual "I Can and I Will" guild run.

“Hailee’s future is bright,” Tiffany said. “She has this ability to light up the room and just radiates joy and happiness. I hope she gets to share that with the world.”

Conclusion

Hailee’s journey is a testament to resilience and hope in the face of adversity. With the support of her family, medical professionals, and the community, she continues to inspire those around her. As she navigates the challenges of living with Morquio A syndrome, her story serves as a reminder of the importance of compassion, understanding, and the power of community in supporting families facing similar struggles.

Resources

• Seattle Children’s Emergency Department
• Mucopolysaccharidosis Type 4A Information
• Seattle Children’s Financial Assistance
• Uncompensated Care Program
• I Can and I Will Guild Run

Hailee’s story is not just about a diagnosis; it’s about the strength of a family and the hope that shines through even the darkest times.