A huge step forward

Since Jack’s diagnosis there have been moments that have been permanently tattooed on me, like the heavy rain pouring down on us when we left the clinic after receiving the news of Jack’s devastating diagnosis, I remember thinking rain harder, I don’t care how hard it rains nothing was ever going to hurt me more than the news we had just received. Since then, we’ve learnt to live with the heartache of watching social media as we have learnt of clinical trial failures and deaths of precious and much-loved members of our community. I don’t forget any of them.

Getting the news that Jack’s trial had positive results is one of those moments that I will never forget. When I got the call, my legs went from under me. I ended up sat on the floor of Asda car park in Wigan looking up at the glorious sun and clear blue skies and I began to cry as I thought of everything that we have been through in the last 10 years. The blood sweat and tears that got us too today. At times I haven’t been able to tell people the true toll it’s taken on us all physically and mentally.

My last blog was about Jack being on a clinical trial and how hard it was to make the decision to put him on. He has been truly incredible. He has taken it all in his stride with a big smile on his face and has willingly complied with every test - there have literally been hundreds. His dad can’t even get into an MRI scanner, but I have lost count of how many times Jack has been in one and has never complained once.

The data the trial has generated has shown that the drug Jack is taking is slowing down the progression of the disease. It’s pushing back the feared milestones of losses we know lie ahead. It’s not a cure but it’s a step in the right direction and everything we have all been working for.

In those tears that fell in Asda car park there was an overwhelming sense of guilt. The knowledge that Jack is 1 of a few hundred boys currently receiving the treatment. There are an estimated 300,000 patients worldwide and 2,500 patients in the UK who will need access to the treatment if future studies are successful.

The trial recruited patients globally, including in the UK through DMD Hub which you helped us fund, and was delivered at four sites funded by Joining Jack and Duchenne UK (Newcastle, Alder Hey, Oswestry and Great Ormond Street Hospital).

For us, we will continue to search for new treatments to help Jack, but this is also the beginning of a new journey to help other people living with Duchenne get access to the drug that’s helping him now. We don’t know how long regulatory approval will take and whether we can get the drug reimbursed but armed with this data set we will help every step of the way to get a treatment for everyone living with Duchenne.

Today, I quite simply want to say thank you to you all for Joining Jack and helping us find a treatment for Duchenne. Please continue to support us on our journey.

Here's the link the press release

Italfarmaco Group Announces Positive Topline Data from Phase 3 Trial Showing Beneficial Effect of Givinostat in Patients with Duchenne Muscular Dystrophy | Business Wire