1. The landscape for rare diseases in 2024;Health;Lancet Glob Health,2024
2. Therapies for rare diseases: therapeutic modalities, progress and challenges ahead;Tambuyzer;Nat Rev Drug Discov,2020
3. RARE Disease Facts. Global Genes. Accessed March 9, 2024. https://globalgenes.org/rare-disease-facts/.
4. 21 USC 360bb: Designation of drugs for rare diseases or conditions. Accessed February 19, 2024. https://uscode.house.gov/view.xhtml?req=200%2C000&f=treesort&fq=true&num=188&hl=true&edition=prelim&granuleId=USC-prelim-title21-section360bb.
5. Designating an orphan product: drugs and biological products;Office of the Commissioner,2022