Rare Disease Day isn’t about numbers. It’s about people—the individual and their family whose world is forever shifted by a diagnosis most have never heard of. It’s about those facing a disease so rare that doctors may struggle to diagnose it, where available treatment options are few, or do not exist, and where research is scarce.
For those living with Metachromatic Leukodystrophy (MLD), every day is a battle against time. It’s a fight for access to specialised care, for treatments that can slow or stop progression, and for the chance to hold on to abilities that MLD threatens to take away. It’s the weight of knowing that solutions may come too late—or may not exist at all.
At Release Tx, we recognise that rare disease isn’t just a statistic. It’s personal. Every diagnosis represents a child, a family, a story that requires us to step up. And every undiagnosed case or misdiagnosis delays critical intervention that affects patients and their families in ways that many cannot imagine.
Today, we stand in solidarity with every person navigating the uncertainty of a rare disease. We are committed to science that doesn’t just push boundaries, but makes a real difference—one life at a time.
Because behind every rare disease is a person who deserves more.
The Release Tx Team
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