Duchenne Muscular Dystrophy

Graeme Downie Excerpts

Thursday 12th June 2025 Commons Chamber

[Chris McDonald (Stockton North) (Lab)]

I thank the Minister for what I think is her triple shift at the Dispatch Box today.

A few months ago, I met three really great young lads—Benjamin, Eli and Jack. We had a great conversation, and they had me laughing and joking along with them for a very long time. I slightly disappointed Eli when I first opened my mouth because, listening to me, he recognised where I was from, and he asked if I could introduce him to Sam Fender. The north-east is quite small, but I do not know everyone, and I could see the disappointment on his face. One of the things we talked about was what they want to do in the future. Eli is considering being a musician, and Jack said he would like to do something involving public speaking or, who knows, even be a politician. It was really great to see that level of ambition, but for me it was tinged with sadness; I knew that they were unlikely to realise their ambitions, because Benjamin, Jack and Eli are unlikely to see their 30th birthdays.

Benjamin, Jack and Eli all have Duchenne muscular dystrophy. It is a disease that affects only boys, and about 100 baby boys are born with it every year. Diagnosis is normally at the age of three or four, and at that moment the parents find out that their son has a life-limiting condition that cannot be treated, and that he will probably need a wheelchair by the age of 12 and a ventilator by the age of 20. There is no cure for Duchenne muscular dystrophy, but there is hope now with a new drug called givinostat.

Givinostat was developed by an Italian company, Italfarmaco, and it is an example of the brilliance of our UK life sciences sector that it decided to develop the drug here in the UK. As it has been trialled here, Italfarmaco has given the national health service a unique option to prescribe givinostat through an early-access programme entirely free of charge. The NHS can offer this drug to boys in the UK completely free of charge, and the early-access programme has been available since November last year. Members might think that such an opportunity would be seized quickly by the NHS, but sadly it has not been. Many families have had their hopes raised, only for them to be dashed when they find that they cannot access the treatment. Patients, families and charities are now calling for urgent access to givinostat, as are many Members of this House.

My constituent Tracy is one of the many who have been fighting for their child’s right to live, and for the treatment that has been proven to slow down the progression of Duchenne. Her son, Tom, is 14 years old, and she tells me that he is deteriorating. Givinostat has market authorisation for all boys with Duchenne over the age of six. It has been proven to slow its progression in clinical trials. It is not a cure—the families realise it is not a cure—but it could help boys and young men to keep walking and use their muscles for longer.

[Graeme Downie (Dunfermline and Dollar) (Lab)]

Like my hon. Friend, I have a constituent, Jamie Tierney, who, sadly, suffers with Duchenne muscular dystrophy. Recently, he has been able to begin treatment, as have other patients in Scotland, but it took a lot of work and in some cases intervention by lawyers to get that. Jamie’s family tell me that “Time is muscle”. Does my hon. Friend agree that the sooner we make givinostat available across all parts of the UK, the better it will be for those people?

[Chris McDonald]

Yes, I do agree with that very timely intervention. In fact, the slogan of Duchenne UK is “Time is muscle”, and it is absolutely right that every day and every week makes a difference to these boys.