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Abstract

Current efforts to foster research on orphan diseases are focused largely on pharmaceutical treatments. Currently, there are roughly 770 FDA approved pharmaceutical treatments designated for orphan diseases. The number of treatments has greatly improved and it can largely be attributed to government efforts to foster research on orphan diseases. However, more can be done to bridge the gap between the number of available treatments, both pharmaceutical and non-pharmaceutical, and the number of different orphan diseases. These efforts should be supplemented by initiatives to bolster diffusion and commercialization of innovations made by the user-innovator community.

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