Hi, we are Cassie and James, proud parents to Elsie.

We never imagined our journey would lead us here. Like any parent, we were simply navigating the everyday joys and challenges of raising our handsome son, Harry, and our beautiful daughter, Elsie. Dreaming of a happy and healthy future for them both.

Then, everything changed.

In 2024 Elsie was diagnosed with Charcot-Marie-Tooth disease type 4J (CMT4J)

This is a rare and progressive genetic condition that affects the peripheral nerves, causing muscle weakness and wasting. Over time, it can lead to wheelchair dependence, quadriplegia, respiratory complications, and a shortened life expectancy.

As a family, hearing those words brought heartbreak, fear, and countless questions about what the future would hold for our little girl.

Elsie is now 13 years old. Your typical teenager! She loves music, reading, playing the keyboard and baking.

Elsie has scoliosis of the spine and struggles to walk unaided. She now relies on mobility aids, a crutch, a walker and a wheelchair for when she is really fatigued. She has to wear AFOs to help with foot drop and she constantly struggles with pain and fatigue on a daily basis.

As parents, watching her go through these struggles is heartbreaking.

But despite everything, Elsie continues to show incredible strength, courage, and resilience every single day. She inspires us constantly, and because of her, we refuse to give up hope.

Elsie has recently taken part in a Natural History Study in the US and now researchers in the United States are preparing for a groundbreaking gene therapy trial for CMT4J later this year.

This treatment aims to deliver a healthy copy of the faulty gene directly to affected cells, with the hope of slowing, stopping, or even reversing the progression of this devastating disease.

For families like ours, this hope means everything. What once felt impossible now feels within reach.

But we have a long journey ahead of us and we need your help.

Developing this life-changing treatment is incredibly expensive. Each single copy of the gene therapy costs between $500,000 and $1 million to produce, and due to funding, only 8 copies of the gene therapy have currently been made to start the first trial.

We are raising money to support CureCMT4J, a nonprofit charity founded by a determined mother whose own daughter also lives with CMT4J and for the last ten years, she has worked tirelessly with scientists and researchers to help make a cure possible.

There is no guarantee that Elsie will be choosen for the first trial but every donation, no matter how big or small makes it possible to develop more copies of the gene therapy and this brings us one step closer to giving Elsie the chance of having this life changing treatment.

Please can you help support us in raising funds to help give our daughter the life and future she deserves ❤️

One family.

One mission.

To help cure CMT4J.