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Rare Disease Day 2025: Reflecting on a year of progress and the challenges that lie ahead
Rare Disease Day presents a time to reflect on the progress made in understanding and addressing the unique challenges that rare diseases bring – and the hurdles that remain. Over the past year, several significant developments have shaped the rare disease landscape, from new regulatory policies and technological advancements to major advocacy efforts.
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The role of precision epidemiology in R&D decision-making: Spotlight on rare genetic diseases
Rare genetic diseases are challenging to treat due to their complexity, the unique nature of each condition, and the – by definition – small numbers of patients affected by each one. Encouragingly for the rare disease community, the field is moving rapidly with a wide variety of innovative therapies being developed, including gene therapy involving […]
