Rare disease awareness is about a brighter future for all of us

As Rare Disease Awareness Month gets underway, it’s a good time to reflect on health conditions that are too often overlooked, misunderstood, and forgotten. Rare Disease Day itself occurs on the last day of February, but for families living with them, awareness isn’t limited to a single day. Rather, it’s a daily reality.

As a Batten disease mom, I know firsthand that rare disease awareness isn’t symbolic; it is lived. It shapes every rhythm of our days and nights, through endless appointments, medication schedules, and the quiet moments when fear and hope coexist in ways most people never witness.

CLN2 disease, also known as late infantile Batten disease, is rare, progressive, and life-limiting. With no cure, it robs children of the skills they once had and the futures they deserve. Its impact extends beyond one child, reshaping entire families. Yet, despite its severity, Batten disease remains largely unknown outside the community it touches. This is why awareness matters so deeply.

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Collective advocacy

When people hear the word “rare,” they often think it means uncommon or a remote possibility. But rare doesn’t feel distant when it lives in your own home. It isn’t abstract when it bears your child’s name. Some 400 million people live with a rare disease globally, and behind every statistic is a family navigating systems never designed for them.

Awareness leads to earlier diagnoses, fuels research, and shapes funding and policy decisions. It opens doors to new treatments, clinical trials, and support services that families desperately need. Without awareness, rare disease families are left fighting alone in spaces where their voices too often go unheard.

For Batten disease families, awareness can mean the difference between access and denial. In CLN2 disease, enzyme replacement therapies exist — but access to them isn’t guaranteed. Families fight for insurance approvals, travel long distances for infusions, and restructure their entire lives around care. Awareness helps others understand that treatment isn’t optional; it’s about survival.

Rare Disease Awareness Month reminds us that no rare disease exists in isolation. Our challenges intersect, and our advocacy is stronger when it is collective.

Living with a rare disease is more than a medical journey — it’s emotional, financial, and deeply personal. Parents become experts overnight. We learn to speak medical language, coordinate care across specialties, advocate within school systems, and challenge decisions that put our children at risk. We grieve losses while still showing up every day with love and determination.

There is a unique isolation that comes with a rare disease. When no one around you has heard of your child’s diagnosis, it can feel profoundly lonely and overwhelming. Awareness builds community. It connects families. It reminds us that, even if our diagnoses are rare, our experiences of love, fear, resilience, and hope are universal.

Batten disease isn’t just a diagnosis. It’s a child’s laughter at their favorite song, a sibling developing compassion far beyond their years, a family discovering how to find joy in moments others might overlook, and a parent learning to hold gratitude and grief in the same breath.

Rare Disease Day is also about honoring the children and adults we have lost. Their lives mattered. Their stories continue to matter. Their legacies inspire research, advocacy, and an ongoing push for better outcomes for future families.

But awareness must also lead to action. Legislative advocacy is critical in shaping the future for rare disease communities. Policies dictate research funding, insurance protections, newborn screening programs, and access to therapies. When rare disease voices are missing from these conversations, decisions are made without our needs in mind.

Advocacy does not require expertise or perfection. It starts with learning the name of a legislative bill, sending an email, sharing your story, or simply showing up — even when it feels uncomfortable. Lawmakers remember stories. They remember families. They remember children.

If you are a rare disease parent reading this, know that your voice holds power. You do not have to advocate alone, nor do you have to do everything at once. Every step forward matters.

For Batten disease families, awareness is how we remain seen. It is how we push for continued research, equitable access to care, and a future where rare no longer means overlooked. February reminds us that while awareness has a date, advocacy never ends.

Rare disease awareness isn’t just about a day or a month — it’s a commitment to keep fighting, together.

Note: Batten Disease News 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 Batten Disease News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to Batten disease.