Why Advocacy Is Important for New Zealanders With Rare Diseases
Columnist Jaime Christmas continues her advocacy in honor of her late husband
Life is full of twists and turns. I was a caregiver for nine years until this past May, when my husband passed away from hereditary ATTR amyloidosis.
Aubrey was diagnosed in 2013, and despite the many challenges we both faced, we never once thought to give up. Unfortunately, due to the thickening of his heart and the multisystemic nature of the disease, we both lost the fight.
Here in New Zealand, many treatment options, such as the gene-silencing therapies Tegsedi (inotersen) and Onpattro (patisiran), are unavailable. Medication to slow the formation of amyloid deposits is available in places like the U.S. and the U.K., while liver transplantation is still the only approved treatment option for New Zealanders diagnosed with hATTR amyloidosis. Yet many amyloidosis specialists now acknowledge that a new liver doesn’t stop disease progression, as patients still get amyloid deposits in other organs.
Since Aubrey’s passing, I find myself pulled toward my work as a patient advocate with the New Zealand Amyloidosis Patients Association. Founded by both of us in 2019, the association has accomplished so much that it’s difficult for me to give it up, despite the change in my situation.
The legacy my late husband left behind grips me with a sense of responsibility. I’m too invested to turn away from the many New Zealanders diagnosed with this illness. But it’s a rough ride. Some days I’m optimistic that we’ll successfully make funded treatments available, while other days I feel like giving up because it’s too hard.
Rare disorders need much more awareness, particularly in this country, where people tend to assume that rare means few. According to Rare Disorders New Zealand, 300,000 people live with a rare condition in our nation. Accessing medications is wrought with challenges, as many modern treatments have yet to be approved here and aren’t funded.
As an advocate, I spend most of my time creating awareness campaigns and finding educational opportunities to speak about the issues rare disease communities face. At the forefront is making doctors aware of amyloidosis and how to identify it. Early diagnosis is essential to ensure longevity and quality of life. This involves a collaborative effort between disease experts and patient advocates.
Despite some setbacks, I’m fortunate to have like-minded and enthusiastic people working alongside me at our association. As British adventurer Bear Grylls said, “My work is all about adventure and teamwork in some of the most inhospitable jungles, mountains and deserts on the planet. If you aren’t able to look after yourself and each other, then people die.”
At the heart of every successful endeavor are people who have stepped up in one way or another to encourage, assist, and lead. Our patient association is no different. I wouldn’t be able to carry on my work after my husband’s passing without the encouragement of the people around me. Step by step, little by little, I’m confident that we’ll all achieve something life-changing in this country for the people we represent.
Note: FAP News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of FAP News Today or its parent company, BioNews, and are intended to spark discussion about issues pertaining to familial amyloid polyneuropathy.