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Volume 6, Issue 1

Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 6, Issue 1 (2018)

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Expert Opinion on Orphan Drugs, Volume 6, Issue 1 (2018)

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Vol 12, 2024 Vol 11, 2023 Vol 10, 2022 Vol 9, 2021 Vol 8, 2020 Vol 7, 2019 Volume 6, 2018 Vol 5, 2017 Vol 4, 2016 Vol 3, 2015 Vol 2, 2014 Vol 1, 2013

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Original Research

Article

Initial combination therapy for patients with pulmonary arterial hypertension (PAH): a budget impact analysis from the perspective of the Italian national healthcare system

Marco Barbieri, Stefano Ghio, Michele D’Alto, Carlo Albera, Renato Carignola, Massimiliano Mulè, Patrizio Vitulo, Miriam Vighini, Rosaria Silvestri, William Zamboni & Carmine Dario Vizza

Pages: 1-7

Published online: 07 Dec 2017

AbstractforInitial combination therapy for patients with pulmonary arterial hypertension (PAH): a budget impact analysis from the perspective of the Italian national healthcare system | Full Text | References | PDF (643.7 KB)

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Review

Mucopolysaccharidosis type III: current clinical trials, challenges and recommendations

Stephanie CM Nijmeijer & Frits A Wijburg

Pages: 9-15

Published online: 07 Dec 2017

AbstractforMucopolysaccharidosis type III: current clinical trials, challenges and recommendations | Full Text | References | PDF (589.3 KB)

128 Views
6 CrossRef citations
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Review

Emerging and potential treatment options for sarcoidosis

Debabrata Bandyopadhyay & Marc A. Judson

Pages: 17-33

Published online: 12 Dec 2017

AbstractforEmerging and potential treatment options for sarcoidosis | Full Text | References | PDF (1.3 MB)

285 Views
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Review

Classification and recommended treatment options for folliculotropic mycosis fungoides

Suzanne van Santen, Maarten H. Vermeer & Rein Willemze

Pages: 35-45

Published online: 27 Nov 2017

AbstractforClassification and recommended treatment options for folliculotropic mycosis fungoides | Full Text | References | PDF (1 MB)

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Review

A developing portrait of hereditary periodic fevers in childhood

Donato Rigante

Pages: 47-55

Published online: 21 Nov 2017

AbstractforA developing portrait of hereditary periodic fevers in childhood | Full Text | References | PDF (722.1 KB)

38 Views
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Review

Emerging therapeutics for the treatment of Friedreich’s ataxia

Elisabetta Indelicato & Sylvia Bösch

Pages: 57-67

Published online: 29 Nov 2017

AbstractforEmerging therapeutics for the treatment of Friedreich’s ataxia | Full Text | References | PDF (941.6 KB)

331 Views
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Review

Is there a role for inhaled anti-inflammatory drugs in cystic fibrosis treatment?

Zara Sheikh, Hui Xin Ong, Michele Pozzoli, Paul M Young & Daniela Traini

Pages: 69-84

Published online: 28 Nov 2017

AbstractforIs there a role for inhaled anti-inflammatory drugs in cystic fibrosis treatment? | Full Text | References | PDF (1.4 MB)

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Drug Evaluation

Article

The role of ibrutinib in Waldenström macroglobulinemia

Maria Gavriatopoulou, Evangelos Terpos, Efstathios Kastritis & Meletios Athanasios Dimopoulos

Pages: 85-89

Published online: 15 Dec 2017

AbstractforThe role of ibrutinib in Waldenström macroglobulinemia | Full Text | References | PDF (396.7 KB)

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