Isaac was three when he was diagnosed on 1 July 2018.

He was lucky enough to enrol on to the vamorolone trial at age four, which was running from the Duchenne Hub UK in Glasgow (Established in partnership between Duchenne UK and the John Walton Muscular Dystrophy Research Centre at Newcastle University). 

When he was seven we were called (through the Duchenne Hub UK database) to ask if Isaac would want to take part in the Pfizer CIFFREO gene therapy trial through the DMD Hub UK in Newcastle. This involved him switching from vamorolone to traditional steroids (in Isaac’s case, prednisolone). We went for the screening, and were lucky enough to be enrolled on to the trial in the nick of time (two days before he was eight).

It was not a smooth road – the trial started and stopped a few times along the way due to serious adverse events globally. Isaac also contracted chicken pox at a critical time in the trial which caused another delay.

Finally, Isaac was dosed with the therapy in November 2023. Shortly thereafter the trial was terminated by Pfizer as it did not meet its primary or secondary endpoints.

Since Isaac was diagnosed, Duchenne UK and everything it has done – through parent information days, conferences, webinars, Duchenne Hub UK and trial sites – has been a huge support to us. We are committed to doing everything we can to treat this condition and support our boys.

We will do this by supporting Duchenne UK, its vision, and the projects it funds.