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Volume 4, Issue 6

Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 4, Issue 6 (2016)

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Expert Opinion on Orphan Drugs, Volume 4, Issue 6 (2016)

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Editorial

The opportunities and challenges of biosimilar orphans

Hoss A. Dowlat

Pages: 563-566

Published online: 07 Apr 2016

First Page PreviewforThe opportunities and challenges of biosimilar orphans | Full Text | References | PDF (307.9 KB)

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Free Access

Original Research

Article

Cost-effectiveness analysis of newborn screening for sickle-cell disease in Spain

Iván Castilla-Rodríguez, Elena Cela, Laura Vallejo-Torres, Cristina Valcárcel-Nazco, Elena Dulín, Mercedes Espada, Dolores Rausell, Javier Mar & Pedro Serrano-Aguilar

Pages: 567-575

Published online: 28 Apr 2016

AbstractforCost-effectiveness analysis of newborn screening for sickle-cell disease in Spain | Full Text | References | PDF (1.3 MB) | Supplemental

143 Views
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Review

Existing therapies and therapeutic targets for erythropoietic protoporphyria

Elisabeth I. Minder, Jasmin Barman-Aksoezen, Michèle Nydegger & Xiaoye Schneider-Yin

Pages: 577-589

Published online: 26 Apr 2016

AbstractforExisting therapies and therapeutic targets for erythropoietic protoporphyria | Full Text | References | PDF (1.2 MB)

64 Views
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Review

Biomarkers and progress of antioxidant therapy for rare mitochondrial disorders

Lucia Chico, Daniele Orsucci, Annalisa Lo Gerfo, Letizia Marconi, Michelangelo Mancuso & Gabriele Siciliano

Pages: 591-603

Published online: 28 Apr 2016

AbstractforBiomarkers and progress of antioxidant therapy for rare mitochondrial disorders | Full Text | References | PDF (963.3 KB)

71 Views
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Review

An update on the pathophysiology, treatment and genetics of Marfan syndrome

Charissa A. C. Jessurun, Debby A. M. Bom & Romy Franken

Pages: 605-612

Published online: 12 May 2016

AbstractforAn update on the pathophysiology, treatment and genetics of Marfan syndrome | Full Text | References | PDF (786.9 KB)

7146 Views
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Open Access

Review

Measures to combat rare diseases and promote orphan drug development in Japan: government-funded special biomedical research programs to enhance basic and applied research

Qi Tang, Peipei Song & Yu Chen

Pages: 613-619

Published online: 02 May 2016

AbstractforMeasures to combat rare diseases and promote orphan drug development in Japan: government-funded special biomedical research programs to enhance basic and applied research | Full Text | References | PDF (663.5 KB)

131 Views
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Review

Biologic therapies in systemic juvenile idiopathic arthritis

Ezgi Deniz Batu

Pages: 621-629

Published online: 28 Apr 2016

AbstractforBiologic therapies in systemic juvenile idiopathic arthritis | Full Text | References | PDF (712.3 KB)

85 Views
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Review

Clinical features and therapeutic strategies for managing the striated muscle laminopathies

Coline Macquart, Rabah Ben Yaou, Antoine Muchir, Karim Wahbi & Gisèle Bonne

Pages: 631-638

Published online: 03 May 2016

AbstractforClinical features and therapeutic strategies for managing the striated muscle laminopathies | Full Text | References | PDF (878.4 KB)

30 Views
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Review

Diagnosis of cardiac involvement in systemic amyloidosis by state-of-the-art echocardiography: where are we now?

Derliz Mereles, Matthias Aurich, Sebastian Greiner, Johannes Riffel, Sebastian J. Buss, Arnt V. Kristen & Hugo A. Katus

Pages: 639-648

Published online: 28 Apr 2016

AbstractforDiagnosis of cardiac involvement in systemic amyloidosis by state-of-the-art echocardiography: where are we now? | Full Text | References | PDF (1.8 MB)

39 Views
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Review

Gene therapy for cystic fibrosis: recent progress and current aims

Michael C. Paul-Smith, Robyn V. Bell, William E. Alton, Eric W.F.W. Alton & Uta Griesenbach

Pages: 649-658

Published online: 06 May 2016

AbstractforGene therapy for cystic fibrosis: recent progress and current aims | Full Text | References | PDF (919.7 KB)

494 Views
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Drug Evaluation

Article

Regorafenib for treatment of imatinib- and sunitinib-resistant metastatic gastrointestinal stromal tumors

Molly M. Daughety & Michael C. Heinrich

Pages: 659-670

Published online: 09 May 2016

AbstractforRegorafenib for treatment of imatinib- and sunitinib-resistant metastatic gastrointestinal stromal tumors | Full Text | References | PDF (1 MB)

62 Views
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Article

Masitinib for gastrointestinal stromal tumors

Mehdi Brahmi & Philippe A. Cassier

Pages: 671-676

Published online: 11 May 2016

AbstractforMasitinib for gastrointestinal stromal tumors | Full Text | References | PDF (501.9 KB)

60 Views
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Article

Deferasirox: an orphan drug for chronic iron overload in non-transfusion dependent thalassemia syndromes

Racha Halawi, Irene Motta, Ali Taher & Maria Domenica Cappellini

Pages: 677-686

Published online: 02 May 2016

AbstractforDeferasirox: an orphan drug for chronic iron overload in non-transfusion dependent thalassemia syndromes | Full Text | References | PDF (860.7 KB)

65 Views
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Expert Opinion on Orphan Drugs (Vol.4, Iss.6, 2016)

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