Orphan Drug Policies: Implications for the United States, Canada, and Developing Countries
Overview
Affiliations
Pharmaceutical policy and innovation for rare diseases: A narrative review.
Ruiz A, Large K, Moon S, Vieira M F1000Res. 2024; 12:211.
PMID: 38778810 PMC: 11109548. DOI: 10.12688/f1000research.130809.2.
Alewaidat H, Bataineh Z, Bani-Ahmad M, Alali M, Almakhadmeh A F1000Res. 2023; 12:741.
PMID: 37822316 PMC: 10562777. DOI: 10.12688/f1000research.130388.1.
Cardiac amyloidosis and surgery: What do we know about rare diseases?.
Mestres C, Van Hemelrijck M J Card Surg. 2021; 36(8):2911-2912.
PMID: 33993557 PMC: 8359931. DOI: 10.1111/jocs.15630.
Regulatory environment for novel therapeutic development in mitochondrial diseases.
Hirano M, Berardo A, Barca E, Emmanuele V, Quinzii C, Simpson C J Inherit Metab Dis. 2020; 44(2):292-300.
PMID: 33368420 PMC: 9326497. DOI: 10.1002/jimd.12353.
The urgent need to empower rare disease organizations in China: an interview-based study.
Li X, Lu Z, Zhang J, Zhang X, Zhang S, Zhou J Orphanet J Rare Dis. 2020; 15(1):282.
PMID: 33046132 PMC: 7552513. DOI: 10.1186/s13023-020-01568-5.