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Volume 3, Issue 6

Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 3, Issue 6 (2015)

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Expert Opinion on Orphan Drugs, Volume 3, Issue 6 (2015)

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Review

Treating limbal stem cell deficiency: current and emerging therapies

Alok Sati (Corneal Surgeon) MS, Sachin Shukla (DST-INSPIRE Faculty, Professor Brien Holden Eye Research Centre) PhD, Ikeda Lal (Cornea Fellow) & Virender S Sangwan (Dr. Paul Dubord Chair in Cornea, Director) MS

Pages: 619-631

Published online: 16 Apr 2015

AbstractforTreating limbal stem cell deficiency: current and emerging therapies | Full Text | References | PDF (606.8 KB)

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Novel therapeutic strategies for the management of ventricular arrhythmias associated with the Brugada syndrome

Bence Patocskai MD & Charles Antzelevitch PhD FHRS FACC FAHA

Pages: 633-651

Published online: 13 May 2015

AbstractforNovel therapeutic strategies for the management of ventricular arrhythmias associated with the Brugada syndrome | Full Text | References | PDF (1.2 MB)

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The road to cure in multiple myeloma starts with smoldering disease

Karma Z Salem MD & Irene M Ghobrial MD

Pages: 653-661

Published online: 12 Apr 2015

AbstractforThe road to cure in multiple myeloma starts with smoldering disease | Full Text | References | PDF (215.9 KB)

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Molecular basis of primary hyperoxaluria and strategies for diagnosis

Gill Rumsby PhD FRCPath

Pages: 663-673

Published online: 12 Apr 2015

AbstractforMolecular basis of primary hyperoxaluria and strategies for diagnosis | Full Text | References | PDF (385.6 KB)

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Recombinant adeno-associated virus vectors in the treatment of rare diseases

Eric Hastie (Postdoctoral Researcher) & R Jude Samulski (Director)

Pages: 675-689

Published online: 15 May 2015

AbstractforRecombinant adeno-associated virus vectors in the treatment of rare diseases | Full Text | References | PDF (313.1 KB)

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Complement inhibition for paroxysmal nocturnal hemoglobinuria: where we stand and where we are going

Antonio M Risitano (Assistant Professor of Hematology, Head of Bone Marrow Transplantation Clinical Unit) MD PhD

Pages: 691-704

Published online: 25 May 2015

AbstractforComplement inhibition for paroxysmal nocturnal hemoglobinuria: where we stand and where we are going | Full Text | References | PDF (417.2 KB)

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Diagnostic tools and strategies for assessing disease progression in Alkaptonuria

LR Ranganath, EE Psarelli, TF Cox & JA Gallagher

Pages: 705-717

Published online: 04 May 2015

AbstractforDiagnostic tools and strategies for assessing disease progression in Alkaptonuria | Full Text | References | PDF (708.3 KB)

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Orphan Drug Designation

Fresh from the designation pipeline: orphan drugs designated in the European Union (November – December 2014)

Sabina Antonela Antoniu MD PhD

Pages: 719-725

Published online: 10 May 2015

AbstractforFresh from the designation pipeline: orphan drugs designated in the European Union (November – December 2014) | Full Text | References | PDF (177.4 KB)

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Drug Evaluation

Miltefosine, an FDA-approved drug for the ‘orphan disease’, leishmaniasis

Jonathan Berman MD PhD

Pages: 727-735

Published online: 25 May 2015

AbstractforMiltefosine, an FDA-approved drug for the ‘orphan disease’, leishmaniasis | Full Text | References | PDF (263.6 KB)

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Ramucirumab for the treatment of gastroesophageal cancers

Yusuke Shimodaira, Elena Elimova, Roopma Wadhwa, Hironori Shiozaki, Nikolaos Charalampakis, Venkatram Planjery, Mariela A Blum, Jeannelyn S Esteralla, Jane E Rogers, Shumei Song & Jaffer A Ajani

Pages: 737-746

Published online: 25 May 2015

AbstractforRamucirumab for the treatment of gastroesophageal cancers | Full Text | References | PDF (333.2 KB)

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Pixantrone dimaleate for treating non-Hodgkin’s lymphoma

Ruth Pettengell & Jasvinder Kaur

Pages: 747-757

Published online: 18 May 2015

AbstractforPixantrone dimaleate for treating non-Hodgkin’s lymphoma | Full Text | References | PDF (286.1 KB)

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Expert Opinion on Orphan Drugs (Vol.3, Iss.6, 2015)

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