Help fund gene therapy to save little Freddie!

Freddie was born May 2021, our perfect bundle of joy, baby no 2. He completed our little family.

At 18 months we noticed Freddie was starting to regress in his motor skills and had become less vocal. He started to lose his balance and core strength.

Months of waiting for an appointment we finally saw a paediatric doctor who expressed major concerns and referred us to Great Ormond Street Hospital.

More months passed, test after test, agonisingly waiting for results and being no closer to any answers.

Until one day genetics got involved.

Nov 2023, Freddie was diagnosed with a rare, 1 in a million, degenerative condition called INAD.

Infantile Neuroaxonal Dystrophy.

We were told there was no cure, and life expectancy was 5-10 years old.

Our world fell apart.

During this time, we were told there was ongoing research into a gene therapy for Freddie’s condition and that it had been going on for some time. Over a decade!

Feb 2024 the research wasn’t as far along as we had hoped.

Fast forward to this year, they have made great progress and but unfortunately it’s now coming down to funding. Because it is such a rare condition, and the current economic climate, funding is very difficult to secure.

Today, Freddie has been stripped of all his motor skills, he can no longer sit, roll, crawl. Loosing his ability to swallow, a feeding tube has now been fitted. He is non verbal with very little understanding.

This condition is so cruel.

Cure INAD is a U.K based non profit charity organisation that has been helping raise funds for the UK gene therapy research and programme.

They are working tirelessly to raise funds to get to the end of the gene therapy programme and run a clinical trial.

It has the potential to stop the progression of this awful condition. A chance for Freddie to fight back.

Please donate what ever means you can, every penny counts. But if you are unable to donate, please share, it’s just as important. We need to get the word out, there is a potentially life changing treatment for Freddie and other children who are running out of time and it’s being delayed due to funding!

INADcure foundation is a US non profit organisation who are also running a gene therapy programme .

They have set up movement called #workingonamiracle to help raise the funds needed to bring it to clinical trial.

Funds may also be used assisting Freddie with any potential medical expenses including potential medical treatment overseas.

Thank you

Emily ( Freddie’s mum)