In November, 2015, Joseph, Melbourne, turned four.
Like any other four-year-old boy, Joe is full of energy and enjoys playing with his big sisters, having play dates, and generally being a little boy.
However, unlike many children his age, in August 2015, at the age of three, Joe was diagnosed with the ultra-rare, life-threatening blood disease, atypical Haemolytic Uraemic Syndrome (aHUS).
Joe’s diagnosis came at a time, when the only clinically prove, life-saving medicine available to treat aHUS, Soliris (eculizumab) was available, and following a quick diagnosis, Joe immediately started treatment with Soliris.
“Joe has been going really well. The medicine has been great for him, and he’s been really healthy,” said Joe’s mum, Sarah.
“Since his diagnosis, which came about very quickly, Joe was granted access to Soliris, and it has been a truly life-saving medicine for him.”
Today, Joseph and Sarah visit Monash Children’s Hospital in Melbourne, once a fortnight for Joe’s Soliris treatment.
“We always keep our fingers crossed that we can just continue the two-week treatment, and hospital visits for Joe. It’s funny, because Joe is so young, the hospital visits are like an outing for him, and he kind of enjoys them. He sees his doctors, and the same nurses every fortnight, and a lot of the same kids,” Sarah said.
“If you didn’t know that Joe was sick, you would never guess at it today, as he is so happy and healthy.
“Because he was so young when he was diagnosed, and because he was able to access Soliris so quickly, his kidney’s are doing really well, and he has bounced back so much since his diagnosis,” said Sarah.
Recognising the need people living with aHUS have for Soliris, Sarah hopes that Joseph’s story is one that helps to demonstrate the amazing benefit that the treatment offers for sustaining and saving the lives of people living with this devastating disease.
“Joe was one of the lucky ones, who was diagnosed when the medicine was available, so he was straight on the medicine, which helped to mitigate any issues. He has no kidney damage, and he is really well and healthy.
“Today, I almost feel like there is nothing wrong with him – he’s fine. We just make sure we manage him, and make sure he gets the medicine every two weeks. In fact, he’s just been reapproved, following his 20-week review for continued access to Soliris, as the medicine has been working really well for him,” Sarah said.
Joseph will be reassessed in August, but at this stage, his kidney levels are good, and he hasn’t been ill as a result of treatment or from his illness. The Soliris is managing Joe’s aHUS, and he is able to enjoy his life, as a happy, healthy four year old.
“We are so thankful that Joe was able to get access to Soliris, and that the doctors are becoming more and more aware of what aHUS is, and how to diagnose it,” said Sarah.
“The aPSGA has done an amazing job in ensuring that its members are made aware of aHUS, and in fighting for Soliris to be funded.
“It’s so sad when we look at what is happening in other countries, where aHUS patients aren’t able to access Soliris. I’ve seen a couple of stories from families who can’t access Soliris for kids a similar age to Joe, and it breaks my heart,” Sarah said.
“It must be so tough having to battle like that, and not get access. We are privileged here, and really do hope that everyone around the world who lives with aHUS, gets access to this amazing, life-saving medicine soon.”