Important Update on Alex’s Clinical Trial

June 30, 2022 11:49 am

This is great news for the Duchenne community. We would like to share this news with all of our wonderful supporters.

We’ve been saying for a while now that Alex is doing incredibly well. Now we know why this might be…….Alex has been taking part in a clinical trial study called Givinostat run by Italfarmaco up in Newcastle for the past 4 years. We suspected he was on the ‘placebo’ arm for the first two years but was moved over to the extension trial two years ago where we were told he would receive the drug. All we have ever wanted since Alex’s diagnosis was a treatment for him that will help slow down disease progression – and the devastating muscle wasting. To buy us more time for the all important cure to come along. We have worked so incredibly hard to make sure he’s seen by the best doctors in the UK. We have worked so tirelessly fundraising for effective treatments to emerge for everyone affected by this awful condition. And on Sunday morning, when I first looked at my phone before our Supercars event – I saw the amazing news that had been published about Alex’s trial.

The data is looking good – it’s showing that it is slowing down disease progression!!!! ♥♥♥♥ I cannot put into words how this makes us feel. To know he’s on a drug that is helping – buying him more time. To know that those dreaded stages we were told about could now be delayed is the most incredible news a parent could ever wish for. It’s a not a cure, we know that, but this is the news that the whole of the Duchenne community has been waiting for. This brings bundles of hope to families. We are incredibly proud of Alex, how he takes everything in his stride. He’s had countless hospital visits, blood tests, MRI scans, physio tests and above all he takes this drug twice a day and he says it tastes horrendous. But this is all a small price to pay. With just 179 boys on the trial worldwide and 300K boys in the world living with Duchenne, we feel incredibly honoured and lucky to have the drug in our fridge.

To know that Alex is helping in his own way to bring effective treatments to the Duchenne community melts my heart. This is such a big step in the right direction. We won’t know how long regulatory approval will take, but with the data they have collected they have everything they need to get going with this. Let’s just hope every single child living with Duchenne gains access to this drug soon. If we can help with that in any way, we will!!The trial recruited patients globally, including in the UK through DMD Hub which Alex’s Wish supports. There is still so much more that needs to be done, to bring effective treatments to market and to find a cure we all so desperately need. Our work continues. We would like to thank all our wonderful supporters from the bottom of our hearts for being on this incredible journey with us. Without your support, there would be no clinical trials, no new drugs and no new medical research taking place. If this shows one thing, its that investing in medical research – does save lives. Thank you. To find out more read on……/Italfarmaco-Group…

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