For MPS Awareness Week this year, we are looking at chasing the signs of MPS and raising awareness of the disease. Sam Brown was diagnosed with MPS IVA Morquio when he was 17 months old. After the recent success of NICE approving Vimizim for MPS IVA, his mother Katy Brown shares what their journey has been like.
“Sam is 13. He loves gaming, Lego and Marvel. He is funny, caring and incredibly headstrong. I can’t get him out of bed on a school day and I’m forever picking socks up, just like most other teenage boys. But unlike most other teenagers, Sam has had multiple surgeries, weekly drug infusions, hospital appointments, school disruption, illness and disability to deal with for pretty much the whole of his life. We are so proud of him and how he deals with these challenges.
I was 32 weeks pregnant with Sam’s younger brother, Alex, when I got the phone call to ask if we’d be interested in Sam taking part in a clinical trial of a new treatment called Vimizim. I said yes immediately, put the phone down and then thought “how on earth are we going to do this?” But not doing it was not an option and as with everything else, we found a way to make it work. Five weeks later, we made the first of our weekly visits to Royal Manchester Children’s Hospital, something that quickly became a family day out for over 4 years when Alex was born 3 weeks later.
Sam has now received Vimizim for a decade. Our weekly eight-hour rounds trips to Manchester are now replaced by me administering his infusion after school at home, slotting it into our busy lives in a way that really works for us. Despite the commitment involved throughout the last decade, the challenges it has created for family life and the impact to Sam of regular medical intervention, the benefits have been significant. Sam’s energy levels know no bounds, he has very little pain and remains independent and mobile.
Keep Sam Smiling:
Back in 2015, we set up the “Keep Sam Smiling” campaign, knowing that we needed to do something to help the MPS Society and the rest of the Morquio community in the fight to get Vimizim approved. I could never envision a world where Sam and others wouldn’t receive this drug; it seemed so wrong. Sam was 6 and quickly became a media star; the twinkle in his eye and a wry smile touched the hearts of the public and generated significant support.
Throughout the experience, Sam had a bus named after him, recorded a pop video, met celebrities like Michael Sheen, and appeared on TV and in the newspapers. We did our best to protect him and make it fun and constantly worried that we were doing the right thing. So, to then hear the news last month that Vimizim has finally been approved by NICE, seven years later, made our hearts sing, and know that it really was all worthwhile in the end.
Sam summed it up brilliantly “It was a hard-fought battle and I enjoyed campaigning for something so important to me. I really enjoyed going to places like Downing Street and making the Keep Sam Smiling music video with my friend. Huge thank you to everyone who helped make this wish come true.”
The pictures include the “Keep Sam Smiling” campaign outside of Parliament and Sam with Michael Sheen.
To donate to our virtual bucket for Awareness Week, please click here.