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Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 2, Issue 10 (2014)

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Expert Opinion on Orphan Drugs, Volume 2, Issue 10 (2014)

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Original Research

Article

Ambrisentan for the treatment of pulmonary arterial hypertension: a budget impact analysis in the Italian context

Marco Barbieri MSc, William Zamboni MD, Lara Pippo MSc, Prateek Madaan BE & Carlo Campana MD

Pages: 989-997

Published online: 11 Aug 2014

AbstractforAmbrisentan for the treatment of pulmonary arterial hypertension: a budget impact analysis in the Italian context | Full Text | References | PDF (304.2 KB)

128 Views
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Reviews

Review Article

An overview of current and future treatment options for patients with cytomegalovirus retinitis

James P Dunn (Associate Professor of Ophthalmology) MD

Pages: 999-1013

Published online: 28 Aug 2014

AbstractforAn overview of current and future treatment options for patients with cytomegalovirus retinitis | Full Text | References | PDF (305.2 KB)

88 Views
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Review Article

An overview of current and future treatment options for Sturge–Weber syndrome

Anne M Comi MD

Pages: 1015-1025

Published online: 29 Sep 2014

AbstractforAn overview of current and future treatment options for Sturge–Weber syndrome | Full Text | References | PDF (466.7 KB)

63 Views
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Review Article

Clinical management of aggressive fibromatosis/desmoid tumour

Marco Maruzzo, Charlotte Benson, Aisha Miah & Ian Judson

Pages: 1027-1036

Published online: 08 Aug 2014

AbstractforClinical management of aggressive fibromatosis/desmoid tumour | Full Text | References | PDF (260.3 KB)

51 Views
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Review Article

Genetics and biological therapies for Alport syndrome

Clifford E Kashtan (Professor of Pediatrics, Director) (Director) MD FASN

Pages: 1037-1047

Published online: 06 Aug 2014

AbstractforGenetics and biological therapies for Alport syndrome | Full Text | References | PDF (323.6 KB)

156 Views
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Review Article

Diagnosis and genetics of Marfan syndrome

Romy Franken, Thomas J Heesterbeek, Vivian de Waard, Aeilko H Zwinderman, Gerard Pals, Barbara JM Mulder & Maarten Groenink MD PhD

Pages: 1049-1062

Published online: 18 Aug 2014

AbstractforDiagnosis and genetics of Marfan syndrome | Full Text | References | PDF (466.6 KB)

479 Views
16 CrossRef citations
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Review Article

Therapeutic strategies for treatment of pulmonary lymphangioleiomyomatosis

Vera P Krymskaya PhD MBA FCPP

Pages: 1063-1074

Published online: 27 Aug 2014

AbstractforTherapeutic strategies for treatment of pulmonary lymphangioleiomyomatosis | Full Text | References | PDF (285.5 KB)

102 Views
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Drug Evaluations

Daunorubicin for the treatment of acute myeloid leukemia

Clara Bertuzzi MD, Stefania Paolini MD PhD, Giuseppe Visani MD & Pier Paolo Piccaluga MD PhD

Pages: 1075-1087

Published online: 21 Aug 2014

Abstractfor Daunorubicin for the treatment of acute myeloid leukemia | Full Text | References | PDF (380.5 KB)

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Pomalidomide for the treatment of relapsed and refractory multiple myeloma

Anna Guidetti MD, Claudia Paba Prada MD, Jacob P Laubach MD, Cindy Varga MD, Michelle E Maglio, Mary McKenney NP, Deborah Doss RN OCN, Robert L Schlossman MD, Constantine Mitsiades MD PhD, Teru Hideshima MD PhD, Güllü T Görgün PhD, Irene M Ghobrial MD, Noopur Raje MD, Nikhil Munshi MD, Kenneth C Anderson MD & Paul G Richardson MD

Pages: 1089-1108

Published online: 06 Sep 2014

Abstractfor Pomalidomide for the treatment of relapsed and refractory multiple myeloma | Full Text | References | PDF (434 KB)

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Recombinant factor VIII Fc (Biogen/Swedish Orphan Biovitrium) for the treatment of hemophilia A

Susannah N Eyre-Brook (Haematology Registrar) BA BMBCh & Mark P Smith (Haematologist) MB ChB FRACP FRCPA

Pages: 1109-1116

Published online: 01 Sep 2014

Abstractfor Recombinant factor VIII Fc (Biogen/Swedish Orphan Biovitrium) for the treatment of hemophilia A | Full Text | References | PDF (423.8 KB)

100 Views
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Research Article

Article

Page: i

Published online: 29 Sep 2014

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Expert Opinion on Orphan Drugs (Vol.2, Iss.10, 2014)

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