02/23/2026
This is another staggering reason why Rare Disease Awareness matters!
Rare diseases may be rare, but their impact is anything but. They shape the lives of children, families, and entire communities across the globe.
While Rare Disease Day is a powerful opportunity to celebrate our global community, the resilience of families, and the progress being made in research and care, it is also a moment to pause and reflect. For millions of families, rare diseases have brought profound hardship and, in too many cases, heartbreaking loss. Today, we honour not only those living with rare conditions, but also the children and loved ones we carry in memory. Their stories deepen our commitment and remind us why this work cannot wait.
It is never easy to speak about death, especially when it concerns children. Yet for far too many families of children born with a rare disease, this remains a devastating reality. Despite important medical advances, one third of children born with a rare disease will not live to see their fifth birthday. Behind this statistic are families navigating uncertainty, limited information, delayed diagnoses, and unequal access to care.
The tools to change this trajectory already exist. Newborn screening programmes can enable earlier diagnosis and timely intervention. Breakthrough research and innovative therapies are offering unprecedented hope. But hope alone is not enough.
Across the world, too many children still lack access to diagnosis, specialized care, and life-saving treatments — often due to cost, limited availability, or fragile health systems.
This is why implementing the Global Action on Rare Diseases (GAPRD) is so critical. By working together — governments, healthcare leaders, researchers, industry, and patient advocates — we can strengthen health systems, expand access to screening and treatment, and ensure that innovation reaches every child who needs it.
Every child deserves the chance to grow, thrive, and live a full life — regardless of how rare their condition may be.
Join us in driving action so that children living with rare diseases are no longer left behind. Because rare diseases affect us all — and together, we can change their story.
