Incredible News! Funding for Treatment Secured & Next Steps!!!

We are writing this with tears of joy and a huge weight lifted from our shoulders. We have some incredible news: Daisy has been officially awarded the funding for her gene therapy treatment and her travel costs to America! This is the miracle we’ve been praying for, and it wouldn't have been possible without the awareness and support you’ve all shown so far. At the moment Daisy is being considered for the National History Study in Iowa. Hopefully when the clinical trial is up and running, Daisy will considered for this too. We are keeping everything crossed and trying to get everything into place ready!

Why we are still fundraising:

While the "big" costs are covered, the journey to get Daisy there—and keep our family stable during this time—is still a mountain to climb. We are shifting our focus to cover the remaining essential costs that the medical funding doesn't include:

*Specialised Medical Travel Insurance: Because of the complexity of Daisy’s condition, this is a significant and mandatory expense for the trip.

• Daily Living & Recovery Costs: Ensuring Daisy has everything she needs while we are in the US for her treatment and recovery.

• Supporting our Home: As Daisy’s needs have increased, I have stepped into a full-time carer role and am no longer able to work. We need a small safety net to ensure our home remains stable and the bills are paid while we focus entirely on Daisy’s health.

We are so close to the finish line. We’ve changed our campaign title to ‘Daisy’s CMT4J Journey: Helping Her Family Reach the Cure!’ to reflect this new chapter.

If you could share this update or donate even a small amount to help us bridge this final gap, it would mean the world to our family. We are almost there!

With love and gratitude,

Adele, Daisy and all of our family ❤️

Hi I’m Adele, (Daisy’s Mum)

in December, we were devastated to find out that our, bright, funny beautiful 14-year-old daughter, Daisy, has CMT4J (Charcot-Marie-Tooth Type 4J) an ultra rare, progressive neurological disease that damages the nerves causing muscle weakness and loss of mobility especially in the hands and feet. Amongst lots of other things, this means losing the ability to walk. The respiratory muscles can also weaken leading to respiratory failure and ventilation. Unlike more common forms of CMT, type 4J is aggressive. For Daisy, this means every day is a challenge, and time is of the essence.

The Hope: A Genetic Trial In American!

we have been given a glimmer of hope that was once thought impossible. There is a groundbreaking genetic treatment trial currently taking place in the United States. This treatment is the best chance we have to stop the progression of this awful disease and give Daisy a future where she can maintain what independence she has left.

However as a family in the UK, the costs of accessing this are astronomical. Between international travel, specialised medical stays and the logistics of navigating a trial overseas, we simply cannot do this alone.

Due to the progression of Daisy’s condition, I have had to leave my job in order to provide 24/7 care, making our financial situation much more difficult.

How you can help.

we are asking for your help to give Daisy a fighting chance. Your donations will go directly towards:

.*Travel and medical expenses for the gene therapy treatment and the Natural History study in the USA.

*Unforeseen costs we will be facing as a family including home adaptations not covered by any funding.

Every pound donated, every share of this page, every word of support means the world to us.

CMT4J may be rare, but our hope is not. We are asking for your help to turn that hope into a reality.

Thank you all so much.