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Volume 2, Issue 1

Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 2, Issue 1 (2014)

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Expert Opinion on Orphan Drugs, Volume 2, Issue 1 (2014)

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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 Vol 3, 2015 Volume 2, 2014 Vol 1, 2013

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Editorial

Overcoming challenges in the acquisition of biospecimens for rare diseases

Elizabeth J Horn (Principal) PhD MBI & Helen M Moore (Acting Chief) PhD

Pages: 1-4

Published online: 22 Nov 2013

AbstractforOvercoming challenges in the acquisition of biospecimens for rare diseases | Full Text | References | PDF (136.9 KB)

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Free Access

Reviews

Review Article

Glanzmann’s thrombasthenia – newer treatment options

Kanjaksha Ghosh & Shrimati Shetty

Pages: 5-10

Published online: 09 Nov 2013

AbstractforGlanzmann’s thrombasthenia – newer treatment options | Full Text | References | PDF (190.2 KB)

74 Views
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Review Article

Treatment of primary biliary cirrhosis

Raoul Poupon (Professor) & Christophe Corpechot

Pages: 11-25

Published online: 12 Dec 2013

AbstractforTreatment of primary biliary cirrhosis | Full Text | References | PDF (336.1 KB)

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Review Article

Canakinumab in autoimmune and autoinflammatory rheumatic diseases

Sabeeda Kadavath MBBS & Petros Efthimiou (Associate Professor of Clinical Medicine) MD FACR

Pages: 27-35

Published online: 06 Dec 2013

AbstractforCanakinumab in autoimmune and autoinflammatory rheumatic diseases | Full Text | References | PDF (327.7 KB)

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Review Article

Therapy options for retinitis pigmentosa

Mitsuru Nakazawa MD PhD

Pages: 37-52

Published online: 22 Nov 2013

AbstractforTherapy options for retinitis pigmentosa | Full Text | References | PDF (529.3 KB)

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Review Article

Pharmacologic management of tuberous sclerosis complex-associated subependymal giant cell astrocytomas

David Neal Franz MD

Pages: 53-66

Published online: 16 Dec 2013

AbstractforPharmacologic management of tuberous sclerosis complex-associated subependymal giant cell astrocytomas | Full Text | References | PDF (479.4 KB)

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Review Article

Alveolar echinococcosis: evaluation of therapeutic strategies

Dominique A Vuitton MD PhD & Solange Bresson-Hadni MD PhD

Pages: 67-86

Published online: 20 Dec 2013

AbstractforAlveolar echinococcosis: evaluation of therapeutic strategies | Full Text | References | PDF (602.4 KB)

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Drug Evaluations

Delamanid, a new 6-nitro-2,3-dihydroimidazo[2,1-b]oxazole for the management of tuberculosis resistant to at least isoniazid and rifampicin

Andreas H Diacon MD PhD, Florian von Groote-Bidlingmaier MD & Peter R Donald MD

Pages: 87-94

Published online: 25 Nov 2013

Abstractfor Delamanid, a new 6-nitro-2,3-dihydroimidazo[2,1-b]oxazole for the management of tuberculosis resistant to at least isoniazid and rifampicin | Full Text | References | PDF (238.3 KB)

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The role of the farnesyltransferase inhibitor lonafarnib in the treatment of Progeria

Mark W Kieran MD PhD, Leslie B Gordon MD PhD & Monica E Kleinman MD

Pages: 95-105

Published online: 13 Dec 2013

AbstractforThe role of the farnesyltransferase inhibitor lonafarnib in the treatment of Progeria | Full Text | References | PDF (465.9 KB)

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Research Article

Article

Page: i

Published online: 20 Dec 2013

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