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US Capitol Capsule: Rare disease sector matures; FDA keeping pace?

This article was originally published in Scrip

Executive Summary

Companies developing drugs to treat rare diseases – traditionally the biotechs – have matured as a sector, and now it's time for them to "really get comfortable in that skin," because increasingly more attention is being paid to the regulatory pathways for those companies' products and the pricing and reimbursement of the medicines, said Sarepta Therapeutics CEO Chris Garabedian.



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