http://www.ncbi.nlm.nih.gov/pubmed/23536010

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?

Gorzelany JA, de Souza MP.

Source

Laurelwood Biopartners, Boston, MA, 02115, USA.

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.