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Volume 2, Issue 12

Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 2, Issue 12 (2014)

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Expert Opinion on Orphan Drugs, Volume 2, Issue 12 (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

Treatment of Wilson’s disease: our patients deserve better

George J Brewer (Sellner Emeritus Professor of Human Genetics, and Emeritus Professor of Internal Medicine) MD

Pages: 1245-1248

Published online: 29 Oct 2014

First Page PreviewforTreatment of Wilson’s disease: our patients deserve better | Full Text | References | PDF (129.2 KB)

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Free Access

Original Research

Article

Decitabine treatment of extramedullary relapse in one patient with T-ALL transformed from myelodysplastic syndrome after allo-PBSCT

Yong You, Zhaodong Zhong, Jieke Cui, Lingli Zou, Ping Zou & Min Zhang

Pages: 1249-1253

Published online: 08 Nov 2014

AbstractforDecitabine treatment of extramedullary relapse in one patient with T-ALL transformed from myelodysplastic syndrome after allo-PBSCT | Full Text | References | PDF (253.8 KB)

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Reviews

Review Article

Treatment strategies for inclusion body myositis

Maren Breithaupt & Jens Schmidt

Pages: 1255-1265

Published online: 14 Nov 2014

AbstractforTreatment strategies for inclusion body myositis | Full Text | References | PDF (329.2 KB)

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Review Article

Improving treatment outcomes in patients with biliary atresia

Rakesh Kumar Thakur (Senior Clinical Fellow) FRCS (Paeds) & Mark Davenport (Professor of Paediatric Surgery) ChM FRCS (Paeds)

Pages: 1267-1277

Published online: 17 Oct 2014

AbstractforImproving treatment outcomes in patients with biliary atresia | Full Text | References | PDF (259.6 KB)

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Review Article

N-of-1 genomic medicine for the rare pediatric genetic diseases

Laurie D Smith PhD MD & Stephen F Kingsmore MB ChB BAO Dsc FRCPath

Pages: 1279-1290

Published online: 05 Nov 2014

AbstractforN-of-1 genomic medicine for the rare pediatric genetic diseases | Full Text | References | PDF (420.4 KB)

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Review Article

The cellular microenvironment: a new target in the search for cellular and molecular treatment for Dupuytren’s disease

David B O'Gorman & Bing Siang Gan

Pages: 1291-1299

Published online: 06 Nov 2014

AbstractforThe cellular microenvironment: a new target in the search for cellular and molecular treatment for Dupuytren’s disease | Full Text | References | PDF (186.3 KB)

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Review Article

Ultra orphan drugs: the NHS model for managing extremely rare diseases

Edmund Jessop & Sheela Upadhyaya

Pages: 1301-1308

Published online: 24 Nov 2014

AbstractforUltra orphan drugs: the NHS model for managing extremely rare diseases | Full Text | References | PDF (178.8 KB)

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Orphan Drug Designation

Article

Fresh from the designation pipeline: orphan drugs recently designated in the EU (June–July 2014)

Sabina Antonela Antoniu MD PhD

Pages: 1309-1313

Published online: 21 Nov 2014

AbstractforFresh from the designation pipeline: orphan drugs recently designated in the EU (June–July 2014) | Full Text | References | PDF (140.5 KB)

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Drug Evaluations

Article

Beraprost sodium MR for the treatment of pulmonary arterial hypertension

Laurence Dewachter PharmD PhD & Robert Naeije MD PhD

Pages: 1315-1323

Published online: 26 Sep 2014

AbstractforBeraprost sodium MR for the treatment of pulmonary arterial hypertension | Full Text | References | PDF (331.3 KB)

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The role of rosuvastatin in the treatment of pediatric homozygous familial hypercholesterolemia

Marjet JAM Braamskamp MD, Roosje J Roelants, Barbara A Hutten & John JP Kastelein

Pages: 1325-1330

Published online: 09 Sep 2014

AbstractforThe role of rosuvastatin in the treatment of pediatric homozygous familial hypercholesterolemia | Full Text | References | PDF (203.4 KB)

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Lenvatinib for the treatment of radioiodine-refractory follicular and papillary thyroid cancer

Jolanta Krajewska & Barbara Jarzab MD PhD

Pages: 1331-1340

Published online: 23 Sep 2014

AbstractforLenvatinib for the treatment of radioiodine-refractory follicular and papillary thyroid cancer | Full Text | References | PDF (247 KB)

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The MEK inhibitor trametinib for the treatment of advanced melanoma

Douglas B Johnson MD, Megan L Hames PharmD & Jeffrey A Sosman MD

Pages: 1341-1349

Published online: 25 Sep 2014

AbstractforThe MEK inhibitor trametinib for the treatment of advanced melanoma | Full Text | References | PDF (279.4 KB)

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Article

Obeticholic acid for the treatment of primary biliary cirrhosis

Kidist K Yimam & Christopher L Bowlus

Pages: 1351-1358

Published online: 29 Sep 2014

AbstractforObeticholic acid for the treatment of primary biliary cirrhosis | Full Text | References | PDF (242 KB)

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Research Article

Article

Page: i

Published online: 24 Nov 2014

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