1. Rare diseases—avoiding misperceptions and establishing realities: the need for reliable epidemiological data;Groft,2010
2. Orphanet. Homepage. [accessed November 2014].
3. The era of genomic medicine;Davies;Clin Med,2013
4. Cost of illness and economic evaluation in rare diseases;Lopez-Bastida,2010
5. Economic considerations in the provision of treatments for rare diseases;McCabe,2010