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Protein Replacement Therapies for Rare Diseases: A Breeze for Regulatory Approval?

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Science Translational Medicine  27 Mar 2013:
Vol. 5, Issue 178, pp. 178fs10
DOI: 10.1126/scitranslmed.3005007


Protein replacement therapies for rare monogenic diseases have a higher probability of regulatory approval compared with biologics, small molecules, and grant-funded orphan drugs.

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