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US Capitol Capsule: Record approvals, new legislation: Big things happening in rare diseases

This article was originally published in Scrip

Executive Summary

With approvals of drugs in the US to treat orphan conditions hitting an all-time high in 2014, new legislative efforts with new incentives that have the potential to be enacted in 2015 and science moving at an unprecedented pace because of new molecular research capabilities, big things are happening in rare diseases, officials declared at a 27 February conference hosted by the National Institutes of Health (NIH).

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