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Facing orphan adoption challenges

This article was originally published in Scrip

Executive Summary

No one doubts the considerable impact the introduction of orphan drug legislation has had on the development of treatments for very rare diseases. However, this progress would not have been achieved were it not for the alliances that have been created among the various stakeholders. Indeed, greater understanding of the fundamental causes of disease, highly motivated patient advocacy groups and the growing interest of big pharma in a space that was until recently the exclusive reserve of biotechs are having a big impact on the prospects for treating patients with rare diseases.




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