- ClinicalTrials.gov. Interventional Study to Assess Efficacy and Safety of Velmanase Alfa in Patients With Alpha Mannosidosis (SHAMAN). Available at: https://clinicaltrials.gov/ct2/show/NCT04031066. Last accessed: January 21, 2020.
- ClinicalTrials.gov. Learn About Clinical Studies. Available at: https://clinicaltrials.gov/ct2/about-studies/learn. Last accessed: January 21, 2020.
- National Institutes of Health. NIH Clinical Research Trials and You. Available at: https://www.nih.gov/health-information/nih-clinical-research-trials-you/basics. Last accessed: January 21, 2020.
- Borgwardt L, Stensland HM, Olsen KJ, et al. Alpha-mannosidosis: correlation between phenotype, genotype and mutant MAN2B1 subcellular localisation. Orphanet J Rare Dis. 2015;10:70.
- European Medicines Agency. New enzyme replacement therapy to treat rare genetic disorder alpha-mannosidosis in children and adults. Available at: https://www.ema.europa.eu/en/news/new-enzyme-replacement-therapy-treat-rare-genetic-disorder-alpha-mannosidosis-children-adults. Last accessed: January 21, 2020.
- Chiesi. Clinical Trial Transparency and Data Sharing. Available at: https://www.chiesi.com/en/research-and-development/clinical-trial-transparency-and-data-sharing/. Last accessed: January 21, 2020.
- Malm D, Nilssen Ø. Alpha-mannosidosis. Orphanet J Rare Dis. 2008;3:21.
- Bavisetty S, Grody WW, Yazdani S. Emergence of pediatric rare diseases: Review of present policies and opportunities for improvement. Rare Dis. 2013;1:e23579.