Koch J, Mayr JA, Alhaddad B, Rauscher C, Bierau J, Kovacs-Nagy R, Coene KL, Bader I, Holzhacker M, Prokisch H, Venselaar H, Wevers RA, Distelmaier F, Polster T, Leiz S, Betzler C, Strom TM, Sperl W, Meitinger T, Wortmann SB, Haack TB. CAD mutations and uridine-responsive epileptic encephalopathy. Brain. 2017;140(Pt 2):279–86. https://doi.org/10.1093/brain/aww300.
Carvalho, M. Extemporaneously Compounded Oral Medicines in European Hospital Pharmacies. Doctoral thesis, UCL (University College London); 2013.
Aprofarm, IX Symposium: Unification of criteria for paediatric formulations – Programme leaflet. Available at: http://www.formulistasdeandalucia.es/ficheros/9472%20triptico%20Pediatria.pdf Accessed: 21 May 2017.
European Parliament and the Council. Directive 2001/83/EC of 6 November on the community code relating to medicinal products for human use. Off J Eur Communities. 2001;L311:67–128.
European Parliament and the Council. Directive 2004/27/EC of 31 march amending directive 2001/83/EC on the community code relating to medicinal products for human use. Off J Eur Union. 2004;L136:34–57.
Council of Europe (2016) Resolution CM/ResAP(2016)1 on quality and safety assurance requirements for medicinal products prepared in pharmacies for the special needs of patients [Online]. Available: https://www.edqm.eu/sites/default/files/resolution_cm_res_2016_1_quality_and_safety_assurance_requirements_for_medicinal_products_prepared_in_pharmacies.pdf Accessed: 20 May 2017.
European Commission (2016) ‘The rules governing medicinal products in the European Union: EU guidelines for good manufacturing practice for medicinal products for human and veterinary use’, Eudralex, Volume 4. Available at: https://ec.europa.eu/health/documents/eudralex/vol-4_en. Accessed: 20 May, 2017.
PIC (2014) PIC/S Guide to good practices for the preparation of medicinal products in healthcare establishments (PE 010–4) [Online]. Available at: https://www.picscheme.org/layout/document.php?id=156. Accessed: 20 May 2017.
Martinez JLV, Stanescu S, Bustamante SC, Del Rey SJ, Macarron CP, Perez AC. Unrecognized transcutaneous severe salicylate intoxication in an infant. Ped Emerg Care. 2015;31(9):8.
Madan RK, Levitt J. A review of toxicity from topical salicylic acid preparations. J Am Acad Dermatol. 2014;70(4):788–92.
Beverley DW, Wheeler D. High plasma urea concentrations in collodion babies. Arch Dis. Childhood. 1986;61:696–8.
Garty BZ. High plasma urea concentration in babies with lamellar ichthyosis. Arch Dis Child. 1986;61(12):1245–6.
Ramirez MJ, Youseef WF, Romero RG, Martinez JMQ, Gonzalez-Ensenat MA, Vilaplana XS, Cubells CL. Acute percutaneous lactic acid poisoning in a child. Ped Dermatol. 2006;23(3):282–5.
Schwam E. Severe accidental overdose of 4-Aminopyridine due to a compounding pharmacy error. J Emerg Med. 2011;41(1):51–4.
Becker ML, Al Hadithy AFY, Van den Bemt PMLA, Hunfeld NGM. Switching to different genetic medicines: a checklist for safety issues. Eur J Hosp Pharm. 2013;20(2):74–7.
Lutz E, Pauletti G, Carvalho M, Davidson G, Ashworth L, Subramaniam V, Llambí F. The role of compounding in closing therapeutic gaps-abstracts from FIP 2013: when not to compound and considerations before compounding. Int J Pharm Compd. 2014;18(1):8–9.
CompoundingToday: Compounding Formulas. Available at: http://compoundingtoday.com/Formulation/. Accessed: 4 June 2017.
Acofarma: Consulta de Formulación Magistral de Acofarma. Available at: http://www.formulacionmagistral.org/. Accessed: 3 June 2017.
Rowe RC, Sheskey PJ, Cook WG, Fenton ME. Handbook of pharmaceutical excipients. Seventh ed. London: Pharmaceutical Press, Royal Pharmaceutical Society; 2012.
Selmin F, Casiraghi A, Minghetti P. Council of Europe Resolution and Pharmacy Compounding. Hospital Pharmacy Europe Suppl: Purchasing for Safety; 2016. p. 6–9.
Commission notice on the application of Articles 3,5 and 7 of Regulation (EC) No 141/2000 on Orphan Medicinal Products. Official Journal of the European Union 2016/C 424/03 of 18 November 2016.
Minghetti P, Giudici E, Montanari L. A proposal to improve the supply of orphan drugs. Pharmacol Res. 2000;42(1):33–7.
Quartel A, Turbeville S, Lounsbury D. Current therapy for Lambert Eaton myastenic syndrome: development of 3,4-diaminipyridine phosphate salt as first-line symptomatic treatment. Curr Med Res Op. 2010;26(6):1363–75.
Roy S, Descamps F, Planas V, Caudron E, Husson MC, Do B. Cysteamine eyedrops: optimisation of the manufacturing process and meeting safety and efficacy criteria for continuous supply. Int J Clin Pharm. 2011;33(2):378–9.
Traupe H, Burgdorf HC. Treatment of Ichthyosis: there is always something you can do. J Am Acad Dermatol. 2007;57:542–7.
Ndri H, Husson MC, Trouvin JH. Injectable sodium benzoate: uses in France and future prospects. Int J Clin Pharm. 2011;33(2):340–1.
Nunn AJ. Making medicines that children can take. Arch Dis Child. 2003;88(5):369–71.
Jaroslawski S, Azaiez C, Korchagina D, Toumi M. Quantifying the persisting orphan-drug shortage public health crisis in the United States. J Mark Access Health Policy. 2016;23(5):1269473.
Selmin F, Musazzi UM, Cilurzo F, Minghetti P. Alternatives when an autorized medicinal product is not available. Medicine Access @ Point of Care. 2017;1(1):e16–21.
Padhy BM, Gupta YK. Drug repositioning: re-investigating existing drugs for new therapeutic indications. J Postgrad Med. 2011;57(2):153–60.
Davies EH, Fulton E, Brook D, Hughes DA. Affordable orphan drugs: a role for non-for-profit organizations. Br J Clin Pharmacol. 2017;83:1595–601.
Dooms M, Pincé H, Simoens S. Do we need authorized orphan drugs when compounded medications are available? J Clin Pharm Ther. 2013 Feb;38(1):1–2.
Simoens S, Cassima D, Picavet E, Dooms M. Are some orphan drugs for rare diseases too expensive? A study of Purches versus compounding costs. Drugs Ther Perspect. 2011;27(10):24–6.
Heemstra H, Cornips MCA, de Meijer M, Rietdijk CD, Slot TK, Meulenhoff PCW & Leufkens HGM. Hospital pharmacy compounding of orphan drugs. European Association of Hospital Pharmacists 13th conference paper Feb 2008.
Schellekens H, Aldosari M, Talsma H, Mastrobattista E. Making individualized drugs a reality. Nat Biotechnol. 2017;35(6):507–13.
Liaw C-Y, Guvendiren M. Current and emerging applications of 3D printing in medicine. Biofabrication. 2017;9:1–18.