ATTR amyloidosis treatment exists, but for many, it’s not accessible

A transthyretin amyloidosis (ATTR) diagnosis is life-altering. This progressive disease causes abnormal protein deposits to slowly accumulate in structures such as the heart and nerves. Left untreated, ATTR cardiomyopathy (ATTR-CM) can shorten life expectancy to within a few years after diagnosis.

My late husband, Aubrey, was diagnosed with hereditary ATTR in 2013. The shock of his diagnosis was quickly followed by another, quieter realization: that his survival may depend not only on biology, but also on geography.

Across much of the world, ATTR can now be treated with disease-modifying therapies. Drugs such as tafamidis, vutrisiran, and acoramidis have transformed the outlook for patients in countries where these medicines are funded. In places such as Australia, the United Kingdom, the United States, and parts of Europe, physicians can prescribe therapies that slow disease progression, reduce hospitalizations, and extend life.

In Australia, for example, tafamidis has been listed on the Pharmaceutical Benefits Scheme, providing subsidized access to people with ATTR-CM. For those patients, the disease’s trajectory may be changing. But for patients living in countries where these medicines are not publicly funded, the experience can be starkly different.

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The emotional burden of unequal access

Here in New Zealand, patients diagnosed with ATTR discover that therapies used overseas remain out of reach locally.

This creates a unique psychological burden. Fear, uncertainty, and grief become layered with a profound sense of injustice. This is certainly the case for me to this day. Since Aubrey passed away in 2022, I cannot help but feel that had he received targeted treatment early on, he may still be with me today.

Patients learn that ATTR treatments exist, that people elsewhere are receiving them. Yet, access to those treatments depends on financial means or the willingness to relocate.

For some, the options become painfully clear:

• Pay enormous out-of-pocket costs for medication that can exceed $100,000 per year
• Move to another country where the treatment is funded
• Stay put and live with the knowledge that therapies that might slow disease progression are simply unavailable

It’s a stark reminder that the accident of birthplace can shape medical destiny.

The social impact on families

The consequences ripple far beyond the patient. Families suddenly face decisions that few people ever expect to make. Adult children wonder if they should relocate their parents overseas. Partners consider uprooting careers or homes to access care in another country. Some families begin fundraising efforts, hoping that community generosity might bridge a gap that healthcare systems have not. Even when relocation is possible, it carries emotional costs. Leaving behind extended family, culture, and support networks can deepen the sense of isolation that chronic illness already brings.

For many patients, however, relocation is not realistic. Work obligations, financial limitations, immigration barriers, or caregiving responsibilities make moving impossible. These patients often describe living in a state of quiet resignation, knowing that medical progress is happening — somewhere else.

Living with therapeutic distance

Patients in underserved systems often speak of a sense of therapeutic distance. They follow international conferences online. They read about clinical trials, new approvals, and expanding treatment guidelines. They see images of patients in other countries beginning therapies that stabilize the disease. Every breakthrough is simultaneously hopeful and painful.

Yet, despite these realities, many patients continue to advocate, not only for themselves but for those who will be diagnosed after them. Patient registries, support groups, and awareness campaigns have become essential tools in bridging the gap between scientific discovery and real-world access.

The power of advocacy and persistence

History has shown that access to rare disease treatments often changes because patients and families refuse to accept silence. Across the world, patient advocates have reshaped policy, accelerated regulatory review, and pushed governments to reconsider funding decisions for rare conditions. Amyloidosis is no exception. Each new therapy underscores the urgency of conversations about equitable access.

Progress rarely happens overnight, but it does happen. Since starting the New Zealand Amyloidosis Patients Association in 2019, I’ve seen positive change. While our access to treatment still lags, more clinicians are now aware of ATTR.

And when progress happens, it changes everything, not just medically but psychologically. When treatment becomes available, the narrative shifts from managing decline to planning for the future.

A future that is closer than it seems

For patients living with ATTR, especially in countries where treatment access remains limited, the journey can feel deeply unfair. Yet the pace of scientific progress offers real reason for optimism. The pipeline of therapies continues to grow, with new stabilizers, gene-silencing therapies, and potentially curative approaches under development. Each advancement strengthens the case for broader access worldwide.

Equally important, patients’ voices are becoming harder to ignore. The history of rare disease medicine teaches us something powerful: Systems eventually change when patients, families, clinicians, and advocates speak with persistence and clarity.

The geography of medicine may still shape who receives treatment today. But it does not have to define tomorrow.

Note: Amyloidosis 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 Amyloidosis News Today or its parent company, Bionews, and are intended to spark discussion about issues pertaining to familial amyloid polyneuropathy.