1. Why rare diseases are an important medical and social issue;Schieppati;Lancet,2008
2. Rare diseases: what's next?;Remuzzi;Lancet,2008
3. Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products;Official Journal of the European Communities,22012000
4. 4 Committee for Proprietary Medicinal Products. Note for Guidance on Repeated Dose Toxicity. July 2000. Available at http://www.emea.europa.eu/pdfs/human/swp/104299en.pdf (last accessed 21 March 2008).
5. 5 Committee for Proprietary Medicinal Products. Note for guidance on duration of chronic toxicity testing in animals (rodent and non rodent toxicity testing). May 1999. Available at http://www.emea.europa.eu/pdfs/human/ich/030095en.pdf (last accessed 26 November 2008).