Rare childhood diseases: how should we respond?

Zurynski, Y; Frith, K; Leonard, H; Elliott, E · Arch Dis Child · 2008

review · Level V

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Abstract

Paradoxically, rare diseases are common, collectively affecting 6-10% of the population and have a huge impact on patients and families, health services, clinicians and the wider community. Accurate data are required to inform clinical practice, government policy and health service planning. We recommend a national approach, similar to that adopted in the USA and Europe, to support research and promote advocacy and equitable access to services for children with rare diseases.

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