Health News
Rare Disease Day 2025
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Hope for a future in which rare diseases are no longer overlooked
Learn how a small biotech built on a big dream considers underserved patient populations at the heart of its innovation. Although each rare disease may have a small patient population, the aggregate of all rare diseases means that a substantial proportion of people are impacted. Current conservative estimates suggest that 18–30 million people in the...
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The evolving landscape of TTR amyloidosis in 2025
Transthyretin (TTR) amyloidosis presents with a wide range of symptoms, meaning that patients often consult multiple specialists. Unfortunately, delays in diagnosis remain a challenge. Many TTR amyloidosis patients experience symptoms for years before receiving a diagnosis, by which time their condition is often advanced and harder to treat. To address this, a group of clinicians...
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Increasing recognition of ATTR cardiac amyloidosis can improve outcomes
Transthyretin Amyloidosis (ATTR) is a multisystemic condition, typically presenting with heart and/or neuropathy symptoms. It is being increasingly recognised in older people due to heightened awareness and advances in cardiac imaging techniques for diagnosis. Amyloidosis is a general term used to describe a group of diseases where a particular protein ‘misfolds’ and subsequently accumulates into...
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Advancing rare disease research without borders
People affected by rare diseases have many unmet health and social care needs. High-quality research is required to improve diagnosis, treatment and support for patients, families and caregivers. Supporting children and young people Raising Awareness of Rare Disease Throughout All Communities (RARDTAC) and the All Ireland Rare Disease Interdisciplinary Research Network (RAiN; Rare Disease All-Ireland Interdisciplinary...
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Rare disease community can now access an app to accelerate diagnosis
Patients can speed up rare disease diagnosis using a secure digital app that stores their data privately. They can share it with their GP or consultant only if they choose. When a diagnostic journey begins, most patients have no clear idea of what they might be facing. They may be searching for answers about a...
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New national rare disease strategy for Ireland
Ireland’s new Rare Disease Strategy will be shaping better care through patient partnership. Learn how this plan will drive real change for those living with rare diseases. Over the coming weeks, a new national strategy for rare diseases in Ireland will be published. The National Rare Disease Strategy (2025–2030) has brought together the expertise of...
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Building blocks for rare disease clinical trials
Clinical trials offer the possibility of life-changing new therapies. However, numerous factors make trials for rare diseases more difficult to do than those targeting larger groups. There are a range of measures to overcome these — some well-established and others that need commitment and investment. Orphan medicine designation The Orphan Medicine Designation (OMD) was introduced...
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Public awareness for people living with rare diseases
Increasing public awareness is essential for understanding the challenges faced by the rare disease community. Awareness helps to foster acceptance and reduces the stigma that is often associated with rare diseases. Rare disease campaign promotes understanding I Am Number Seventeen is a public awareness campaign initiated and funded by Takeda Products Ireland in partnership with...