Today, on Rare Disease Day, we pause to recognise the millions of individuals and families navigating conditions that too often remain overlooked.
In drug discovery and development, rare diseases remind us why innovation, collaboration, and persistence are essential.
Five facts that underscore the urgency:
🔬 Rare is common: over 300 million people worldwide live with a rare disease.
🧬 Many conditions, limited treatments: more than 7,000 rare diseases are known, yet fewer than 10% have an approved treatment.
👶 Often starts early: around 70% of rare diseases begin in childhood.
🧪 Diagnosis can take years: patients frequently face long diagnostic journeys, delaying care and support.
🤝 Collaboration is critical: progress in rare disease drug development depends on strong partnerships across academia, industry, regulators, and patient communities.
On this Rare Disease Day, we reaffirm our commitment to advancing science where the unmet need is greatest. Every dataset, every assay, and every clinical insight brings us one step closer to meaningful therapies.
#RareDiseaseDay #DrugDiscovery #Biotech #PatientCentricity #Innovation
