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Volume 3, Issue 12

Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 3, Issue 12 (2015)

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Expert Opinion on Orphan Drugs, Volume 3, Issue 12 (2015)

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

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Reviews

Potential of antisense therapy for facioscapulohumeral muscular dystrophy

Bo Bao (Faculty of Medicine and Dentistry) & Toshifumi Yokota (Faculty of Medicine and Dentistry) (Muscular Dystrophy Canada Research Chair)

Pages: 1365-1374

Published online: 24 Sep 2015

AbstractforPotential of antisense therapy for facioscapulohumeral muscular dystrophy | Full Text | References | PDF (573.4 KB)

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A short introduction to acyl-CoA dehydrogenases; deficiencies and novel treatment strategies

Martin Lund (PhD student) MSc, Rikke KJ Olsen (Associate Professor) PhD & Niels Gregersen (Professor emeritus) DMSc

Pages: 1375-1386

Published online: 24 Sep 2015

AbstractforA short introduction to acyl-CoA dehydrogenases; deficiencies and novel treatment strategies | Full Text | References | PDF (525 KB)

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Progress and challenges in gene therapy for Crigler–Najjar syndrome

Sem J. Aronson, Ulrich Beuers & Piter J. Bosma

Pages: 1387-1396

Published online: 11 Nov 2015

AbstractforProgress and challenges in gene therapy for Crigler–Najjar syndrome | Full Text | References | PDF (287.5 KB)

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Therapeutic potential of orphan drugs for the rare skeletal muscle diseases

Kristy Swiderski & Gordon S Lynch

Pages: 1397-1425

Published online: 12 Sep 2015

AbstractforTherapeutic potential of orphan drugs for the rare skeletal muscle diseases | Full Text | References | PDF (887.5 KB)

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Understanding molecular mechanisms in propionic acidemia and investigated therapeutic strategies

Eva Richard (Associate Professor) PhD, Belén Pérez (Associate Professor) PhD, Celia Pérez-Cerdá PhD & Lourdes R Desviat (Associate Professor) PhD

Pages: 1427-1438

Published online: 22 Sep 2015

AbstractforUnderstanding molecular mechanisms in propionic acidemia and investigated therapeutic strategies | Full Text | References | PDF (764.4 KB)

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Advances in therapeutic strategies for Leber’s hereditary optic neuropathy

Rustum Karanjia & Alfredo A. Sadun

Pages: 1439-1446

Published online: 11 Nov 2015

AbstractforAdvances in therapeutic strategies for Leber’s hereditary optic neuropathy | Full Text | References | PDF (294.1 KB)

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Pathophysiology and emerging therapeutic strategies in Pelizaeus–Merzbacher disease

Hitoshi Osaka & Ken Inoue

Pages: 1447-1459

Published online: 29 Oct 2015

AbstractforPathophysiology and emerging therapeutic strategies in Pelizaeus–Merzbacher disease | Full Text | References | PDF (911.3 KB)

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Drug Evaluations

Article

Sapacitabine in acute myelogenous leukemia

James Mccloskey, Jamie Koprivnikar & Stefan Faderl

Pages: 1461-1468

Published online: 02 Nov 2015

AbstractforSapacitabine in acute myelogenous leukemia | Full Text | References | PDF (293.7 KB)

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Article

Cabozantinib: an orphan drug for thyroid cancer

D. Viola, V. Cappagli, A. Matrone, S. Mazzeo & R. Elisei

Pages: 1469-1477

Published online: 20 Nov 2015

AbstractforCabozantinib: an orphan drug for thyroid cancer | Full Text | References | PDF (585.6 KB)

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Article

Droxidopa for neurogenic orthostatic hypotension

William P. Cheshire

Pages: 1479-1490

Published online: 13 Nov 2015

AbstractforDroxidopa for neurogenic orthostatic hypotension | Full Text | References | PDF (314.4 KB)

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Article

Defibrotide for the treatment of severe hepatic veno-occlusive disease/sinusoidal obstruction syndrome: evidence for clinical benefit

Paul G Richardson, Amrita Krishnan, Sergio Giralt & Robert J Soiffer

Pages: 1491-1501

Published online: 23 Nov 2015

AbstractforDefibrotide for the treatment of severe hepatic veno-occlusive disease/sinusoidal obstruction syndrome: evidence for clinical benefit | Full Text | References | PDF (314.5 KB)

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Corrigendum

Correction

Corrigendum

Page: 1

Published online: 11 Dec 2015

CitationforCorrigendum | Full Text | PDF (30.1 KB)

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