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Volume 3, Issue 9

Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 3, Issue 9 (2015)

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Expert Opinion on Orphan Drugs, Volume 3, Issue 9 (2015)

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Editorial

Evaluating a novel treatment for coronary artery inflammation in acute Kawasaki disease: a Phase I/IIa trial of atorvastatin

Adriana H Tremoulet (Associate Professor, Associate Director) MD MAS, Sonia Jain (Professor, Co-Director) PhD & Jane C Burns (Professor, Director) MD

Pages: 967-970

Published online: 13 Jul 2015

AbstractforEvaluating a novel treatment for coronary artery inflammation in acute Kawasaki disease: a Phase I/IIa trial of atorvastatin | Full Text | References | PDF (163.9 KB)

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Editorial

Neonatal screening in Spain and cost–effectiveness

Pedro Serrano-Aguilar MD PhD MPH, Iván Castilla-Rodríguez PhD, Laura Vallejo-Torres PhD, Cristina Valcárcel-Nazco BSc & Lidia García-Pérez BSc

Pages: 971-974

Published online: 31 Jul 2015

AbstractforNeonatal screening in Spain and cost–effectiveness | Full Text | References | PDF (154.4 KB)

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Review

Recent advances in laboratory-aided diagnosis of von Willebrand disease

Emmanuel J Favaloro PhD FFSc (RCPA)

Pages: 975-995

Published online: 10 Jul 2015

AbstractforRecent advances in laboratory-aided diagnosis of von Willebrand disease | Full Text | References | PDF (1,017.1 KB)

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Obstacles and future of gene therapy for hemophilia

Valder R Arruda MD PhD & Ben J Samelson-Jones

Pages: 997-1010

Published online: 18 Jul 2015

AbstractforObstacles and future of gene therapy for hemophilia | Full Text | References | PDF (418.8 KB)

207 Views
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Investigated and available therapeutic options for treating aceruloplasminemia

Hiroaki Miyajima

Pages: 1011-1020

Published online: 13 Jul 2015

AbstractforInvestigated and available therapeutic options for treating aceruloplasminemia | Full Text | References | PDF (699.2 KB)

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New discovered molecular markers as promising therapeutic targets in germ cell tumors

Paolo Chieffi & Francesca Boscia

Pages: 1021-1030

Published online: 28 Jul 2015

AbstractforNew discovered molecular markers as promising therapeutic targets in germ cell tumors | Full Text | References | PDF (665.3 KB)

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Investigative and emerging treatments for Batten disease

Marta A Tarczyluk (Postdoctoral Research Worker) PhD & Jonathan D Cooper (Professor of Experimental Neuropathology) PhD

Pages: 1031-1045

Published online: 27 Jul 2015

AbstractforInvestigative and emerging treatments for Batten disease | Full Text | References | PDF (325.6 KB)

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Globin gene regulation for treating β-thalassemias: progress, obstacles and future

Antoine N Saliba MD, Raafat S Alameddine MD, Afif R Harb MD & Ali T Taher MD PhD FRCP

Pages: 1047-1062

Published online: 24 Aug 2015

AbstractforGlobin gene regulation for treating β-thalassemias: progress, obstacles and future | Full Text | References | PDF (929.8 KB)

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Current therapeutic options for managing familial Mediterranean fever

Ezgi Deniz Batu (Instructor of Pediatrics) , Zehra Serap Arici (Instructor of Pediatrics) , Yelda Bilginer (Professor of Pediatrics) & Seza Özen (Professor of Pediatrics)

Pages: 1063-1073

Published online: 27 Jul 2015

AbstractforCurrent therapeutic options for managing familial Mediterranean fever | Full Text | References | PDF (306 KB)

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Drug Evaluation

Dabrafenib for the treatment of melanoma

Sarah A Weiss MD & Anna C Pavlick DO

Pages: 1075-1084

Published online: 24 Aug 2015

AbstractforDabrafenib for the treatment of melanoma | Full Text | References | PDF (409.6 KB)

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Ruxolitinib for treatment of polycythemia vera

Chad C Cherington, Krisstina L Gowin & Ruben A Mesa MD

Pages: 1085-1096

Published online: 24 Aug 2015

AbstractforRuxolitinib for treatment of polycythemia vera | Full Text | References | PDF (478.9 KB)

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Expert Opinion on Orphan Drugs (Vol.3, Iss.9, 2015)

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