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C-Path Consortium Aims To Speed, De-Risk Duchenne R&D

This article was originally published in Scrip

Executive Summary

While some firms in recent years have found great success with rare disease medicines – with orphan drugs turning out to be the new blockbusters in return on investment – it's nonetheless, generally been a rough road for those products getting to the marketplace, given the difficulty in enrolling patients and proving efficacy in small populations.

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