Charlie was born on January 13, 2019, a bright, spirited little girl who quickly captured the hearts of everyone around her. But just 14 days later, she was diagnosed with MPS1 Hurler Syndrome, a rare, progressive, and life-threatening genetic disorder. This meant Charlie’s body lacked a vital enzyme to break down complex sugars. Without it, these sugars build up in her organs, tissues, and even her brain, causing severe and painful complications over time.

Thanks to newborn screening in Tennessee, Charlie was diagnosed early, allowing her to begin enzyme replacement therapy (ERT) just 30 days after birth. ERT provided a crucial bridge, but it wasn’t enough to protect her brain from the effects of the disease. To save Charlie's life and give her a chance at a future, Charlie and her mom embark on a journey to secure a bone marrow transplant, the most effective treatment currently available for MPS1.

Charlie underwent her first transplant in May 2019. Despite the challenges and terrifying odds, Charlie’s spirit remained unbreakable. Over the next few years, she would bravely endure multiple transplants, surgeries, and countless treatments. Her journey has been filled with both hope and heartbreak, as her mother, Mandy, fought tirelessly to get Charlie the specialized care she needed. From relocating across states to advocating for out-of-state Medicaid approvals, Mandy has relentlessly ensured Charlie has the best possible chance.

Today, Charlie is a joyful five-year-old who loves school, laughs often, and has a big heart for her friends. But MPS1 is a relentless disease that continues to impact her body. Each day brings new challenges and pain, yet Charlie’s resilience shines through. She will likely face more surgeries for her hips, knees, and spine and ongoing treatments to manage the complications of this cruel disease.

With your support, the Kennedy Ladd Foundation can bring hope to Charlie and other children battling MPS1 by advancing research for better treatment options and, ultimately, a cure. Every donation brings us one step closer to a world where children like Charlie can live without the burdens of this devastating disease. Please join us in supporting Charlie’s journey and the mission to find a cure for MPS1. Together, we can bring hope and a brighter future to these incredible children.

We Need Your Help Now!

This is a cry for help. MPS1 Hurler Syndrome is relentless, but so is the fight for a cure. Your donation can fuel groundbreaking research, spark a movement, and bring hope to families like Charlie’s who are in desperate need.Together, we can create a future where MPS1 is no longer a life sentence. Let’s fight for a cure.

Donate today and be part of the solution. Every dollar makes a difference. Help us raise $1,000,000 for The Kennedy Ladd Foundation to fund life-saving research and bring hope to children like Charlie.

Learn more about Blayne's story here

How You Can Help:

Donate Now: Every donation counts and brings us closer to a cure for MPS1.

Share Charlie’s Story: Help us reach more people by sharing Charlie’s story on social media with the hashtag #HopeForCharlie and encouraging others to give.

Ask Your Friends: Ask 10 of your friends to donate $10. A small contribution from many can create a big change.

Join Charlie’s Team: Fundraise with Us!

Charlie needs a crew, and we’re calling on you to suit up and join the mission! By starting your own fundraiser under Charlie’s banner, you’re not just raising funds, you're becoming part of Charlie’s Team—a community dedicated to spreading awareness and fighting to cure MPS1.

Invite your friends, family, and neighbors to rally behind you. Together, we’ll move mountains and take huge strides toward curing MPS1. The more people we have on the team, the louder our voices will echo, and the greater our impact will be.

How to Get Started:

Sign up to create your own fundraiser as part of Charlie’s Team.
Rally your community to join the fight and spread the word.
Watch as we come together to make a difference, one story, and one donation at a time.

Let’s do this—because every superhero needs a super team! 👊

Hope for Charlie: Help Us Cure MPS1 Hurler Syndrome

By Kennedy Ladd Foundation

Hope for Charlie: Help Us Cure MPS1 Hurler Syndrome

Help us CURE MPS1

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