title: Protein replacement therapies for rare diseases: a breeze for regulatory approval?
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.
authors.affiliation: Laurelwood Biopartners, Boston, MA 02115, USA. jen@laurelwoodbio.com
authors.forename: Jennifer A
authors.initials: JA
authors.lastname: Gorzelany
authors.forename: Mark P
authors.initials: MP
authors.lastname: de Souza
mesh_terms: D017277:Drug Approval / Q000331:legislation & jurisprudence*; D056947:Enzyme Replacement Therapy; D006801:Humans; D009965:Orphan Drug Production / Q000331:legislation & jurisprudence; D011506:Proteins / Q000627:therapeutic use*; D035583:Rare Diseases / Q000188:drug therapy*; D014481:United States; D014486:United States Food and Drug Administration
chemical_list: D011506:Proteins