Bibliography
- European Medicines Agency. Public summary of opinion on orphan designation: 2-[4-Methoxy-3-(2-m-tolyl-ethoxy)-benzoylamino]-indan-2-carboxylic acid for the treatment of systemic sclerosis. 2013
- Conron M, Beynon HL. Churg-Strauss syndrome. Thorax 2000;55(10):870-7
- Kim S, Marigowda G, Oren E, et al. Mepolizumab as a steroid-sparing treatment option in patients with Churg-Strauss syndrome. J Allergy Clinical Immunol 2010;125(6):1336-43
- European Medicines Agency. Public summary of opinion on orphan designation-Mepolizumab for the treatment of Churg-Strauss syndrome. 2013
- Allikmets R, Singh N, Sun H, et al. A photoreceptor cell-specific ATP-binding transporter gene (ABCR) is mutated in recessive Starqardt macular dystrophy. Nat Genet 1997;15(3):236-46
- European Medicines Agency. Public summary of opinion on orphan designation: Ramiprilat for the treatment of Stargardt's disease. 2013
- Haltia M. The neuronal ceroid-lipofuscinoses: from past to present. Biochim Biophys Acta 2006;1762(10):850-6
- European Medicines Agency. Public summary of opinion on orphan designation: Recombinant human tripeptidyl-peptidase 1 for the treatment of neuronal ceroid lipofuscinosis type 2. 2013
- Kinali M, Manzur AY, Muntoni F. Recent developments in the management of Duchenne muscular dystrophy. Paediatrics Child Health 2008;18(1):22-6
- European Medicines Agency. Public summary of opinion on orphan designation: R,S-O-(3-piperidino-2-hydroxy-1-propyl)-nicotinic acid amidoxime dihydrochloride for the treatment of Duchenne muscular dystrophy. 2013.