Looking back, the 1983 Orphan Drug Act was a pivotal moment for healthcare, not just in the US but worldwide too. Research on orphan diseases has since flourished, paving the way in 2017 for the first US-approved gene therapy launch, and to date, hundreds of gene therapies are in the pipeline. Jeremy Edwards of Raremark, a patient community online specialist in rare conditions, outlines the challenges and opportunities in the orphan space.

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