2 Office of Orphan Products Development, US Food and Drug Administration, Silver Spring, Maryland 20993, USA.
3 European Medicines Agency, 30 Churchill Place, Canary Wharf, E14 5EU London, UK; and Läkemedelsverket, Dag Hammarskjölds vägen 42, 75103 Uppsala, Sweden.
4 European Medicines Agency, 30 Churchill Place, Canary Wharf, E14 5EU London, UK; and European Organisation for Rare Diseases (Eurordis), 96 rue Didot, 75014 Paris, France.
5 Evaluation and Licensing Division, Pharmaceutical and Food Safety Bureau (PFSB), Ministry of Health, Labour and Welfare (MHLW), 1-2-2 Kasumigaseki Chiyoda-ku, Tokyo, 100-8916 Japan.
6 European Medicines Agency, 30 Churchill Place, Canary Wharf, E14 5EU London, UK; and Universidade de Lisboa, Faculdade de Farmácia, 1649-003 Lisboa, Portugal.