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Since the first orphan druglegislation was passed over 40 years ago, the landscape has changed rapidly, and patients are benefiting more and more from breakthrough innovations emerging through this scientific revolution. What is the current treatment landscape for rare diseases? What is working well and what is lacking?
The expansion of technology and the advancement of new tools have aided drug discovery and essentially brought orphan drugs into a new sphere within the drug market, renewing conversations for needed changes to legislation. Revising legislation.
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