Expert Opinion on Orphan Drugs, Volume 4, Issue 12 (2016)

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Expert Opinion on Orphan Drugs, Volume 4, Issue 12 (2016)

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Editorial

Editorial
Orphan drugs in Australia
Geoffrey K. Herkes
Pages: 1195-1197
Published online: 17 Nov 2016
First Page PreviewforOrphan drugs in Australia | Full Text | References | PDF (291.5 KB)
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Original Research

Article
Individualized therapy in patients with Fabry disease: is it a feasible strategy for rare diseases?
S Manrique-Rodríguez, A de la Rubia-Nieto, JA García-Robles, M Goicoechea-Diezhandino, JA Gimeno-Blanes, MS Pernía-López, C Pellicer-Franco & M Sanjurjo-Sáez
Pages: 1199-1206
Published online: 08 Nov 2016
AbstractforIndividualized therapy in patients with Fabry disease: is it a feasible strategy for rare diseases? | Full Text | References | PDF (599.2 KB)
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Review

Review
Antibodies in autoimmune retinopathy
Pooja Bhat & Siya Huo
Pages: 1207-1216
Published online: 24 Oct 2016
AbstractforAntibodies in autoimmune retinopathy | Full Text | References | PDF (761.6 KB)
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Review
Immune thrombocytopenia: a comprehensive review from pathophysiology to promising treatment modalities
Hassan M. Moukhadder, Bachar F. Chaya, Abdul-Hamid A. Bazarbachi & Ali T. Taher
Pages: 1217-1227
Published online: 26 Oct 2016
AbstractforImmune thrombocytopenia: a comprehensive review from pathophysiology to promising treatment modalities | Full Text | References | PDF (1.1 MB)
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Review
Rare disease: a focus on metabolomics
Desiderio Piras, Emanuela Locci, Francesco Palmas, Giulio Ferino, Vassilios Fanos, Antonio Noto, Ernesto D’aloja & Gabriele Finco
Pages: 1229-1237
Published online: 09 Nov 2016
AbstractforRare disease: a focus on metabolomics | Full Text | References | PDF (1.3 MB)
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Review
Pathogenesis, clinical features and diagnosis of sarcoglycanopathies
Corrado Angelini & Marina Fanin
Pages: 1239-1251
Published online: 17 Nov 2016
AbstractforPathogenesis, clinical features and diagnosis of sarcoglycanopathies | Full Text | References | PDF (1.1 MB)
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Review
Emerging diagnostic and therapeutic options for treating mucormycosis
Matthew W. McCarthy & Thomas J. Walsh
Pages: 1253-1261
Published online: 02 Nov 2016
AbstractforEmerging diagnostic and therapeutic options for treating mucormycosis | Full Text | References | PDF (704.9 KB)
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Review
Therapeutic targets and investigated treatments for Ataxia-Telangiectasia
Martin F. Lavin, Abrey J. Yeo, Amanda W. Kijas, Ernst Wolvetang, Peter D. Sly, Claire Wainwright & Kate Sinclair
Pages: 1263-1276
Published online: 11 Nov 2016
AbstractforTherapeutic targets and investigated treatments for Ataxia-Telangiectasia | Full Text | References | PDF (1.3 MB)
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Drug Evaluation

Article
Mogamulizumab in the treatment of cutaneous T cell lymphoma
Frank Winsett, Xiao Ni & Madeleine Duvic
Pages: 1277-1280
Published online: 30 Nov 2016
AbstractforMogamulizumab in the treatment of cutaneous T cell lymphoma | Full Text | References | PDF (573.8 KB)
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