01/03/2026
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Over 7,000 rare diseases affect millions worldwide.
Yet, for most, there’s no effective treatment. Why? Because research often starts with animal models that don’t reflect human biology.
Rare diseases are usually caused by unique human genetic mutations. Animals don’t share these complexities. When scientists “model” them in mice, the symptoms and progression rarely match what patients experience.
Take, for example: Duchenne Muscular Dystrophy. Drugs that worked in mice failed repeatedly in humans. That's a lot of lost money, time, and hope.
The reality is that species differences matter. Animals simply have different immune responses, different gene expression, and different organ systems, which mean treatments that look promising in animals often fail in clinical trials.
Instead, we need research methods that are actually relevant to humans. Patient-derived cells, organ-on-chip systems, and advanced computational models are ethical alternatives that are paving the way for breakthroughs.
This Rare Disease Day, we're calling for more human-relevant research into these conditions!