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. 2013 Mar 27;5(178):178fs10.
doi: 10.1126/scitranslmed.3005007.

Protein replacement therapies for rare diseases: a breeze for regulatory approval?

Jennifer A Gorzelany  1 , Mark P de Souza
Affiliations

Protein replacement therapies for rare diseases: a breeze for regulatory approval?

Jennifer A Gorzelany et al. Sci Transl Med. .

Abstract

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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