1. National Center for Advancing Translational Sciences and Genetic and Rare Diseases Information Center. In: FAQs about rare diseases. 2016.
https://rarediseases.info.nih.gov/diseases/pages/31/faqs-about-rare-diseases
. Accessed 14 Dec 2018.
2. Drummond MF, Wilson DA, Kanavos P, Ubel P, Rovira J. Assessing the economic challenges posed by orphan drugs. Int J Tech Ass Health Care. 2007;23:36–42.
3. López-Bastida J, Oliva-Moreno J. Cost of illness and economic evaluation in rare diseases. Adv Exp Med Biol. 2010;686:273–82.
4. Prasad S, James E. The challenges associated with developing therapies for rare diseases. Br J Med Pr. 2009;1:42–8.
5. Hirschhorn R, Reuser AJJ. Glycogen storage disease type II: acid α-glucosidase (acid maltase) deficiency. In: Scriver CR, Beaudet AL, Sly WS, Valle D, editors. The metabolic and molecular basis of inherited disease, vol. 3. 8th ed. New York: McGraw-Hill; 2001. p. 3389–420.