When hope gets shattered

A little light of hope was thrown our way when we went to Summit Therapeutics in December 2017 to hear all about their latest trial for DMD. This trial is (was) the only ‘trial’ & ‘shimmer of hope’ on the drugs market for William and his duplication DMD.

That hope has now been lost – the trial has been stopped suddenly today: my heart literally has been ripped out.

You see there are many ways that Duchenne can manifest in a body – William has a duplication of exons 53/54. This means that all other trials on the current market are not applicable for him: we did have some hope.

I don’t know how to explain the immediate loss of hope I felt when I read this today, it was the first time I cried in front of Phoebe and could not stop: I felt sick.

There is no way to say how you feel with a child who has a life limiting disease, no cure and the odd carrot dangled. So far no carrot has come anyway near. I really thought Utrophin might be the one – clearly not.

William’s consultant at our last check up said she would put him through screening for the next phase of Utrophin – this was literally less than a month ago – and now it’s gone.

It was my hope, but many of my DMD mummy friends sons were on this trial already – they found out their sons future on twitter – devastating.

So my break away with the hubby last week – which was lovely and rekindled ‘us’ – feels like a million miles away today. No amount of mini breaks, nights out, holidays and adventures ever takes away the ultimate black cloud on your heart – a cure. One that now seems even further away.

Gutted

As I always say #fightthefight #findacure #smashdmd -but today I’ve not got much strength.

Tomorrow is another day

3 thoughts on “When hope gets shattered

  1. There are a lot of ongoing trials with potential to help William. I realize the options currently available in the UK are far less than in the US, but there is a lot of reason to be hopeful. Gene therapy is especially exciting and there are presently, three different candidates in clinical trials. In theory, the myostatin inhibitor being investigated in this clinical trial could benefit William. https://dmdhub.org/trials/roche-bms-986089/ I believe I read that he’s five though and this study is open to kids six and older. You may want to look into it though if you haven’t already. They plan to enroll 159 boys ages 6-11, which is a lot considering how rare DMD is. They began recruiting in July 2017. I imagine it will be a while yet before it’s fully enrolled.

    Like

    1. Thanks for the message Josh. Yes aware of the gene editing which is exciting. I’m aware of Roche trial but also not done lots due to his age – it may be worth pursuing though if what you say about recruiting is true. Thank you

      Like

Leave a Reply

Fill in your details below or click an icon to log in:

WordPress.com Logo

You are commenting using your WordPress.com account. Log Out /  Change )

Google photo

You are commenting using your Google account. Log Out /  Change )

Twitter picture

You are commenting using your Twitter account. Log Out /  Change )

Facebook photo

You are commenting using your Facebook account. Log Out /  Change )

Connecting to %s