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References
- ↑ "Prescription medicines: registration of new chemical entities in Australia, 2014". Therapeutic Goods Administration (TGA). 21 June 2022. Retrieved 10 April 2023.
- ↑ "FDA-sourced list of all drugs with black box warnings (Use Download Full Results and View Query links.)". nctr-crs.fda.gov. FDA . Retrieved 22 Oct 2023.
- ↑ "FDA approves Vimizim to treat rare congenital enzyme disorder". Food and Drug Administration. Feb 14, 2014. Archived from the original on January 27, 2017. Retrieved December 16, 2019.
- ↑ Hendriksz CJ, Burton B, Fleming TR, Harmatz P, Hughes D, Jones SA, et al. (November 2014). "Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study". Journal of Inherited Metabolic Disease. 37 (6): 979–90. doi:10.1007/s10545-014-9715-6. PMC 4206772 . PMID 24810369.
- ↑ Hendriksz CJ, Giugliani R, Harmatz P, Mengel E, Guffon N, Valayannopoulos V, et al. (February 2015). "Multi-domain impact of elosufase alfa in Morquio A syndrome in the pivotal phase III trial". Molecular Genetics and Metabolism. 114 (2): 178–85. doi: 10.1016/j.ymgme.2014.08.012 . PMID 25284089.
- ↑ "Providing $400,000 life-saving drug to Australian kids with rare disease. The Australian Government will provide a life-saving treatment to Australian patients who have a rare medical condition known as Morquio A Syndrome, at no cost. The Hon Greg Hunt MP. Minister for Health. 15 June 2017". Archived from the original on 14 April 2018. Retrieved 13 September 2018.
- ↑ "BioMarin moet verder levensreddend middel leveren aan patiënte" [BioMarin must continue to provide life-saving drug to patient]. www.tijd.be (in Dutch). De Tijd. 26 June 2019. Archived from the original on 27 June 2019. Retrieved 26 June 2019.