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Volume 4, Issue 2

Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 4, Issue 2 (2016)

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Expert Opinion on Orphan Drugs, Volume 4, Issue 2 (2016)

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Editorial

HDAC inhibitors for muscular dystrophies: progress and prospects

M. Sandoná, S. Consalvi, L. Tucciarone, P. L. Puri & V. Saccone

Pages: 125-127

Published online: 29 Dec 2015

First Page PreviewforHDAC inhibitors for muscular dystrophies: progress and prospects | Full Text | References | PDF (219.7 KB)

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Review

Prospects and progress of oncolytic viruses for treating peripheral nerve sheath tumors

Slawomir Antoszczyk & Samuel D. Rabkin

Pages: 129-138

Published online: 26 Dec 2015

AbstractforProspects and progress of oncolytic viruses for treating peripheral nerve sheath tumors | Full Text | References | PDF (858.5 KB)

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Antisense oligonucleotide development for the treatment of muscular dystrophies

Tri Le Bao, Rakesh N. Veedu, Sue Fletcher & Steve D. Wilton

Pages: 139-152

Published online: 15 Dec 2015

AbstractforAntisense oligonucleotide development for the treatment of muscular dystrophies | Full Text | References | PDF (1.2 MB)

308 Views
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Epidemiological aspects of inhibitor development in hemophilia and strategies of management

Jenny Klintman & Erik Berntorp

Pages: 153-168

Published online: 14 Jan 2016

AbstractforEpidemiological aspects of inhibitor development in hemophilia and strategies of management | Full Text | References | PDF (1.1 MB)

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Prospect of gene therapy for cardiomyopathy in hereditary muscular dystrophy

Yongping Yue, Ibrahim M. Binalsheikh, Stacey B. Leach, Timothy L. Domeier & Dongsheng Duan

Pages: 169-183

Published online: 17 Dec 2015

AbstractforProspect of gene therapy for cardiomyopathy in hereditary muscular dystrophy | Full Text | References | PDF (1 MB)

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Pharmacotherapy options for membranous nephropathy

Arrigo Schieppati & Giuseppe Remuzzi

Pages: 185-196

Published online: 23 Dec 2015

AbstractforPharmacotherapy options for membranous nephropathy | Full Text | References | PDF (886 KB)

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Managing seizures in mitochondrial disorders

Josef Finsterer & Sinda Zarrouk-Mahjoub

Pages: 197-204

Published online: 27 Dec 2015

AbstractforManaging seizures in mitochondrial disorders | Full Text | References | PDF (620.3 KB)

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Histaminergic modulation in Tourette syndrome

Joanna H. Cox, Stefano Seri & Andrea E. Cavanna

Pages: 205-213

Published online: 05 Jan 2016

AbstractforHistaminergic modulation in Tourette syndrome | Full Text | References | PDF (679.2 KB)

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Drug Evaluation

Article

Elotuzumab for treating myeloma

María-Victoria Mateos

Pages: 215-222

Published online: 25 Jan 2016

AbstractforElotuzumab for treating myeloma | Full Text | References | PDF (560.7 KB)

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Article

Selumetinib for the treatment of melanoma

Kimberly M. Komatsubara & Richard D. Carvajal

Pages: 223-231

Published online: 28 Jan 2016

AbstractforSelumetinib for the treatment of melanoma | Full Text | References | PDF (724.2 KB)

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Article

Combination lumacaftor and ivacaftor therapy for cystic fibrosis

Susanna A. McColley

Pages: 233-242

Published online: 12 Feb 2016

AbstractforCombination lumacaftor and ivacaftor therapy for cystic fibrosis | Full Text | References | PDF (1.3 MB)

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