Duchenne Muscular Dystrophy affects one in every 3,500 boys.
Teenager Alex Hallam has been part of a clinical study to treat Duchenne Muscular Dystrophy.
Alex has been part of the study for the last three years, participating in the Phase 3 clinical trial over the last 18 months. The new anti-fibrotic drug, Givinostat, has been shown to have a good tolerability profile and positive outcomes from trial patients, according to latest data released by drug sponsor Italfarmaco.
The company will now meet with regulatory agencies to share these findings and plan how to submit the complete dataset in a marketing application for potential approval.
An aggressive form of muscular dystrophy, Duchenne affects one in every 3,500 boys and there is currently no cure.
Alex’s mum, Emma Hallam, launched the charity, Alex’s Wish, almost 10 years ago to raise funds for medical research and ultimately eradicate Duchenne.
"The latest data from this study is the news the Duchenne community has been waiting for," said Emma. "This provides new hope for boys living with Duchenne, their families, and the medical community. We know it’s not a cure, but we also know Alex is now on a medication that is buying him precious time while we wait for a cure to come along.
“Knowing that those dreadful stages we were told about when our son was diagnosed at age four could now be delayed, is the best news a parent could ever wish for. This is such a big step in the right direction.”
Alex is among just 179 boys on the trial and patients were recruited globally. Alex’s Wish helps support The DMD Hub, which was instrumental in recruiting patients onto this trial in the UK.
As a trial participant, Alex has countless hospital visits, regular blood tests, physio tests, MRI scans and must take Givinostat – a liquid medicine which he says tastes ‘horrendous’ – twice a day. But this is a small price to pay.
Emma added: “To know that Alex is helping in his own way to bring effective treatments to the Duchenne community melts my heart. We feel incredibly honoured and lucky to have the drug in our fridge.
“We won’t know how long regulatory approval will take, but we must now fight to get access to this drug in the UK, so everyone has access to it. We must also continue to find a cure that will halt disease progression in boys living with Duchenne.
“Alex’s Wish would like to thank all our wonderful supporters for being on this incredible journey with us. Without their amazing support, there would be no clinical trials, no new drugs, and no new medical research. The result of this trial clearly shows that investing in medical research does save lives.”
