Protein replacement therapies for rare diseases: a breeze for regulatory approval?
Where this comes from
- Record sourced from PubMed, PMID 23536010.
- Also identified by DOI 10.1126/scitranslmed.3005007.
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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.
Medical subject headings
- Drug Approval
- Proteins
- Rare Diseases