Fresh from the designation pipeline: orphan drugs recently designated in the EU (June–July 2014)

Bibliography

• Antoniu S. Fresh from the designation pipeline: orphan drugs recently designated in the EU (March–April 2014). Expert Opin Orphan Drugs 2014;2(9):957-60
   Google Scholar
• Antoniu S. Fresh from the designation pipeline: orphan drugs recently designated in the European Union (June – August 2013). Expert Opin Orphan Drugs 2013;1(12):1063-71
   Web of Science ®Google Scholar
• Sawamura D, Nakano H, Matsuzaki Y. Overview of epidermolysis bullosa. J Dermatol 2010;37(3):214-19
   PubMedGoogle Scholar
• Woodley DT, Keene DR, Atha T, et al. Injection of recombinant human type VII collagen restores collagen function in dystrophic epidermolysis bullosa. Nat Med 2004;10(7):693-5
   PubMed Web of Science ®Google Scholar
• Agency EM. Public summary of opinion on orphan designation: recombinant human alpha 1 chain homotrimer of type VII collagen for the treatment of epidermolysis bullosa. 2014
   Google Scholar
• Mctaggart KE, Tran M, Mah DY, et al. Mutational analysis of patients with the diagnosis of choroideremia. Hum Mutat 2002;20(3):189-96
   Google Scholar
• Maclaren RE, Groppe M, Barnard AR, et al. Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial. Lancet 2014;383(9923):1129-37
   PubMed Web of Science ®Google Scholar
• Agency EM. Public summary of opinion on orphan designation: adeno-associated viral vector serotype 2 containing the human CHM gene encoding human Rab escort protein 1 for the treatment of choroideraemia. 2014
   Google Scholar
• Agency EM. Public summary of opinion on orphan designation: adeno-associated viral vector serotype 2 containing the human REP1 gene for the treatment of choroideraemia. 2014
   Google Scholar
• Valstar MJ, Neijs S, Bruggenwirth HT, et al. Mucopolysaccharidosis type IIIA: clinical spectrum and genotype-phenotype correlations. Ann Neurol 2010;68(6):876-87
   PubMed Web of Science ®Google Scholar
• Quiviger M, Arfi A, Mansard D, et al. High and prolonged sulfamidase secretion by the liver of MPS-IIIA mice following hydrodynamic tail vein delivery of antibiotic-free pFAR4 plasmid vector. Gene Ther 2014;21
   Google Scholar
• Agency EM. Public summary of opinion on orphan designation: autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene for the treatment of mucopolysaccharidosis IIIA (Sanfilippo A syndrome). 2014
   Google Scholar
• Kremer H, Van Wijk E, Marker T, et al. Usher syndrome: molecular links of pathogenesis, proteins and pathways. Hum Mol Genet 2006;15(2):R262-70
   Google Scholar
• Hashimoto T, Gibbs D, Lillo C, et al. Lentiviral gene replacement therapy of retinas in a mouse model for Usher syndrome type 1B. Gene Ther 2007;14(7):584-94
   PubMed Web of Science ®Google Scholar
• Agency EM. Public summary of opinion on orphan designation: mixture of two adeno-associated viral vectors serotype 8 containing the 5’-half sequence of human MYO7A gene and the 3’-half sequence of humanMYO7A gene for the treatment of Usher syndrome. 2014
   Google Scholar
• Antoniu S. Fresh from the designation pipeline: orphan drugs recently designated in the European Union (September – November. 2013). Expert Opin Orphan Drugs 2014;2(3):311-15
   Web of Science ®Google Scholar
• Agency EM. Public summary of opinion on orphan designation: mixture of two adeno-associated viral vectors of serotye 8 containing the 5’-half sequence of human ABCA4 gene and the 3’-half sequence of humanABCA4 gene for the treatment of Stargardt’s disease 2014
   Google Scholar
• Fabrikant J, Touloei K, Brown SM. A review and update on melanocyte stimulating hormone therapy: afamelanotide. J Drugs Dermatol 2013;12(7):775-9
   Google Scholar
• Agency EM. Public summary of opinion on orphan designation: afamelanotide for the treatment of familial benign chronic pemphigus (Hailey-Hailey disease). 2014
   Google Scholar
• Pepys MB, Herbert J, Hutchinson WL, et al. Targeted pharmacological depletion of serum amyloid P component for treatment of human amyloidosis. Nature 2002;417(6886):254-9
   PubMed Web of Science ®Google Scholar
• Wall JS, Kennel SJ, Williams A, et al. AL amyloid imaging and therapy with a monoclonal antibody to a cryptic epitope on amyloid fibrils. PLoS One 2012;7(12):e52686
   PubMed Web of Science ®Google Scholar
• Agency EM. Public summary of opinion on orphan designation: carboxy pyrrolidine hexanoyl pyrrolidine carboxylate for the treatment of AL amyloidosis. 2014
   Google Scholar
• Agency EM. Public summary of opinion on orphan designation:recombinant monoclonal antibody to human serum amyloid P component for the treatment of AL amyloidosis. 2014
   Google Scholar