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Enzyme replacement therapy: improving outcomes in rare disease

European Pharmaceutical Review

SEPTEMBER 12, 2024

Since the first orphan drug legislation 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?

Drug Legislation 52
Drug Legislation Hospitals 52
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Evaluate’s Orphan Drug Report 2022

pharmaphorum

JUNE 20, 2022

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.

Drug Legislation 52
Drug Legislation 52
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