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

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

Author information

1
Laurelwood Biopartners, Boston, MA 02115, USA. jen@laurelwoodbio.com

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.

PMID:
23536010
DOI:
10.1126/scitranslmed.3005007
[Indexed for MEDLINE]

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