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Volume 4, Issue 10

Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 4, Issue 10 (2016)

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Expert Opinion on Orphan Drugs, Volume 4, Issue 10 (2016)

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Editorial

Suitability of expanding the priority review voucher into rare disease drug development

Sana Mostaghim & Aaron S. Kesselheim

Pages: 1001-1003

Published online: 24 Aug 2016

First Page PreviewforSuitability of expanding the priority review voucher into rare disease drug development | Full Text | References | PDF (288.6 KB)

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Review

Gitelman syndrome: a review of clinical features, genetic diagnosis and therapeutic management

Fernando Santos, Helena Gil-Peña, Cristina Blázquez & Eliecer Coto

Pages: 1005-1009

Published online: 22 Aug 2016

AbstractforGitelman syndrome: a review of clinical features, genetic diagnosis and therapeutic management | Full Text | References | PDF (438.4 KB)

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Review

Targets and investigative treatments for primary biliary cholangitis

Ahmad H. Ali, Elizabeth J. Carey & Keith D. Lindor

Pages: 1011-1020

Published online: 06 Sep 2016

AbstractforTargets and investigative treatments for primary biliary cholangitis | Full Text | References | PDF (762.8 KB)

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Review

Identifying and managing lupus nephritis during pregnancy

Micaela Fredi, Gina Gregorini, Sonia Zatti, Andrea Lojacono & Angela Tincani

Pages: 1021-1031

Published online: 13 Sep 2016

AbstractforIdentifying and managing lupus nephritis during pregnancy | Full Text | References | PDF (833 KB)

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Review

CFTR modulator therapies in pediatric cystic fibrosis: focus on ivacaftor

Elizabeth L. Kramer & John P. Clancy

Pages: 1033-1042

Published online: 13 Sep 2016

AbstractforCFTR modulator therapies in pediatric cystic fibrosis: focus on ivacaftor | Full Text | References | PDF (727 KB)

184 Views
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Review

Neurobiologically-based treatments in Rett syndrome: opportunities and challenges

Walter E. Kaufmann, Jennifer L. Stallworth, David B. Everman & Steven A. Skinner

Pages: 1043-1055

Published online: 10 Sep 2016

AbstractforNeurobiologically-based treatments in Rett syndrome: opportunities and challenges | Full Text | References | PDF (1.3 MB)

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Open Access

Review

Current treatment of Langerhans cell histiocytosis

Chalinee Monsereenusorn, Milen Minkov & Carlos Rodriguez-Galindo

Pages: 1057-1068

Published online: 21 Sep 2016

AbstractforCurrent treatment of Langerhans cell histiocytosis | Full Text | References | PDF (896 KB)

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Review

Therapeutic targets and investigated treatment strategies in Acanthamoeba keratitis

Jacob Lorenzo-Morales, María Reyes-Batlle, Ines Sifaoui, Francisco Arnalich-Montiel, Atteneri López-Arencibia, Carolina Wagner, Pedro Rocha-Cabrera, Antonio del Castillo-Remiro, Enrique Martínez-Carretero, José E. Piñero & Basilio Valladares

Pages: 1069-1073

Published online: 02 Sep 2016

AbstractforTherapeutic targets and investigated treatment strategies in Acanthamoeba keratitis | Full Text | References | PDF (574.5 KB)

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Meta-Opinion

Article

Meta-opinion: from screening to diagnosis of Pompe disease – a European perspective

Zoltan Lukacs & Benedikt Schoser

Pages: 1075-1078

Published online: 01 Sep 2016

AbstractforMeta-opinion: from screening to diagnosis of Pompe disease – a European perspective | Full Text | References | PDF (369.2 KB)

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Drug Evaluation

Article

Extended-release tacrolimus tablets for preventing organ transplant rejection

Timothy A. Horwedel & Daniel C. Brennan

Pages: 1079-1086

Published online: 11 Aug 2016

AbstractforExtended-release tacrolimus tablets for preventing organ transplant rejection | Full Text | References | PDF (863.5 KB)

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Article

Prospect & progress of venetoclax in treating chronic lymphocytic leukemia

Ami Chitalia & Chaitra Ujjani

Pages: 1087-1093

Published online: 08 Sep 2016

AbstractforProspect & progress of venetoclax in treating chronic lymphocytic leukemia | Full Text | References | PDF (646.6 KB)

99 Views
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Corrigendum

Correction

Corrigendum

Page: 1094

Published online: 26 Sep 2016

CitationforCorrigendum | Full Text | PDF (83.5 KB)

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Expert Opinion on Orphan Drugs (Vol.4, Iss.10, 2016)

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