February 29 is the most rare date on the calendar, coming around only once every four years.
This year, in 2016, February 29 marks Rare Disease Day, with the theme “patient voice”.
This Rare Disease Day, people from all around the world are joining together to ensure the voices of those living with rare diseases continue to be heard, voicing the needs of patients living with rare disease, the needs of their families, friends and carers, and working to instigate change.
In 2016, on Rare Disease Day, the aPSGA wanted to reflect on some of the struggles and battles faced in the past 12 months, and to look forward to the year ahead in hopes to continue having our voices heard, and to continue fighting to #saveaHUSlives.
aPSGA President, Kerri Grey says thanks to the efforts of our members, friends, supporters, family and all of you who helped lend your voice to save the lives of those Australians living with aHUS, we have seen access to Soliris (eculizumab) extended for another 12 months for aHUS patients in many cases, and while we continue to strive for all of our members, and all Australians living with aHUS to access Soliris for life, we are pleased to know our voices have been heard, and continue to be heard.
“This Rare Disease Day, reflecting on the year that has passed for the aPSGA, I am so happy that we were able to access to eculizumab extended.
“Further to this, looking ahead, we are hopeful to have a good outcome at the March PBAC meeting for the use of eculizumab in transplant patients living with aHUS,” said Kerri.
“We will continue to voice the needs of our members and keep aHUS on the radar of those in power.
“At some point soon, we will have to continue our fight to stop people coming off treatment,” Kerri said.
“One other thing we really look forward to this Rare Disease Day is awareness of new treatments and we continue to watch the progress of new treatments as they become available.”
Like Kerri, aPSGA treasurer, Debbie Basham is thankful for the work of the aPSGA over the past 12-months, and as she continues her recovery post-kidney transplant, Debbie is excited as doctors are yet to report any signs of aHUS.
“Having a transplant is a wonderful and exciting time, but it can also be very stressful and nerve racking,” said Debbie.
“It’s been seven weeks since my transplant, and so far, there has been no sign of my aHUS, which I am extremely thankful for.
“However, as I begin preparations to leave the hospital in Brisbane, and to go home to Cairns, which I am really excited about, there is a little niggle that something may go wrong so close to home time and every little thing out of the ordinary feels twice as bad as it really is,” Debbie said.
This Rare Disease Day, Debbie is thankful for the support of local communities, and all Australians and people internationally who are working to make sure Rare Diseases are not forgotten about.
“In my local community, in Cairns, they held a walk yesterday in support of Rare Disease Day – I couldn’t take part this year, as I continue my recovery in Brisbane, but I did last year, and I am so impressed that more than 500 people registered to take part in the walk this year, which is fabulous,” Debbie said.
She also voiced her hopes for the year ahead, and like Kerri, Debbie is hoping for a good result at the March PBAC meeting regarding the listing of Soliris for aHUS patients, pre-and post-transplant, which would allow more members of the aPSGA and Australian aHUS community a chance at organ replacement.
“Next month soliris for pre and post transplant patients will go to the PBAC. My fingers are crossed that it will get approved and I will only be the first of many aHUS patients receiving a transplant and having the chance of a normal life,” said Debbie.
“It is so exciting to finally be able to make plans without always considering dialysis. Just having my afternoons free to garden, visit my grandson or to just relax with my husband and have a drink on our deck sounds wonderful.”
To all of our members, friends, family and supporters, we thank you all for continuing on this journey with us, and we hope that on Rare Disease Day 2017, we can report that our voices were heard, and that we are one step closer to helping #saveaHUSlives.