Mandy’s 12-year-old son, TJ, who has Duchenne muscular dystrophy, received Vamorolone for six years via a clinical trial. She believes he gained huge benefits on the drug. In celebration of Vamorolone being recommended by NICE (National Institute for Health and Care Excellence) on 10 December, Mandy tells their story. She shares their personal experience of how they feel Vamorolone helped TJ, and what happened when he came off the treatment and switched to standard use corticosteroids.
“Vamorolone helped my son maintain his independence”
TJ did amazingly on Vamorolone. At age 11, he was still walking and only needed to use his wheelchair for long distances. His medical team would often comment on how great he was doing for someone his age with Duchenne. Then we had to stop Vamorolone and start steroids. Everything changed in an instant. After three months TJ couldn’t walk a single step.
Accessing clinical trials for Duchenne muscular dystrophy
When TJ was diagnosed with Duchenne muscular dystrophy at the age of three, life changed for us as a family. I went from being a mum of three to also being a full-time carer, trying to hold myself together whilst going through the worst heartbreak I could ever imagine.
I was constantly on the lookout for new clinical trials that we could join to try and slow the progression of TJ’s condition.
In 2017, when he was five, TJ started Vamorolone. He was able to experience the benefits of the medication for six years.
The clinical trial was of course intense at first; we had to be at Newcastle hospital, which was a four hour round trip for us, every Monday and Tuesday for the entire day. TJ had to endure countless tests and blood draws, but it was worth it for the benefits we saw. As Vamorolone is an oral drug administered daily, after a while, we were able to have it delivered to our house and only had to go for checkups every now and then.
We know others have experienced side effects with Vamorolone, but other than a little weight gain, which we were able to control with some small diet changes, we were so relieved TJ didn’t.
TJ was on Vamorolone until he was 11. It felt like a miracle that we’d only seen a minimal decline in his condition over the six years from him starting it. He was still able to walk independently around school. He could dress himself and take himself to the toilet. He had as much independence as we could have hoped for since his diagnosis.
Starting steroids and stopping Vamorolone was an impossible decision to make
In March 2023, we made the difficult decision to stop TJ’s Vamorolone and move him on to steroids so that he was able to participate in another clinical trial. We were nervous because we were aware of some possible side effects, but we never imagined his decline would be so rapid.
A couple of days after stopping Vamorolone, TJ began to fall over more frequently.
Within three months of being off it, he couldn’t walk a single step anymore. TJ now needs help with almost everything, from getting in and out of his chair, to getting dressed, to brushing his teeth.
As well as this loss of his independence, it’s been heartbreaking watching the side effects. He’s experienced rapid weight gain, and we’ve tried everything we can think of to slow this down. But exercising in the hot tub, swimming three times a week, and following the strictest possible diet hasn’t helped. For the first time since his diagnosis, TJ has become unhappy and is really upset about the change to his body.
Vamorolone means hope for families like mine
When I found out Vamorolone was going to be recommended by NICE I was ecstatic.
I know treatments like this can affect everyone differently; one of the boys that we were on the clinical trial with experienced a much quicker decline compared to TJ. But what this drug brings, is hope and options.
Until this point, the only available medication for Duchenne on the NHS has been two types of steroids. If neither of them works for an individual, there is no other options but to watch your child deteriorate. Vamorolone gives families another option and hope for a brighter future with, in our experience, less side effects.
Although we were very lucky TJ was able to receive Vamorolone, clinical trials take their toll on children and families like mine. It was amazing to be able to tell TJ that we did it; everything he’d been through had helped make this drug available to thousands of others. All the drives in the dark of the night, all the tears and pain and exhaustion, had been worth it to get to this point.
Read our full announcement
NICE (the National Institute for Health and Care Excellence) has recommended that the drug vamorolone (also sold under the brand name Agamree) be made available for people aged four years and over with Duchenne muscular dystrophy in England. This is a big step forward in the availability of treatment options for the condition and provides an alternative to the corticosteroids that are currently prescribed.
