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Volume 3, Issue 8

Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 3, Issue 8 (2015)

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Expert Opinion on Orphan Drugs, Volume 3, Issue 8 (2015)

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Editorial

The importance of natural histories for rare diseases

Pamela Gavin

Pages: 855-857

Published online: 20 Jul 2015

AbstractforThe importance of natural histories for rare diseases | Full Text | References | PDF (125.5 KB)

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Review

Large granular lymphocyte leukemia: clinical background, molecular pathogenesis and treatment

Francis R LeBlanc & Thomas P Loughran Jr

Pages: 859-867

Published online: 01 Jul 2015

AbstractforLarge granular lymphocyte leukemia: clinical background, molecular pathogenesis and treatment | Full Text | References | PDF (267.1 KB)

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Diagnosis, prevalence, and screening of familial dilated cardiomyopathy

Mary E Sweet BA, Matthew RG Taylor MD PhD & Luisa Mestroni MD

Pages: 869-876

Published online: 22 Jun 2015

AbstractforDiagnosis, prevalence, and screening of familial dilated cardiomyopathy | Full Text | References | PDF (377.1 KB)

255 Views
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Current and investigated alternatives to botulinum toxin for managing blepharospasm

Roberta Pellicciari & Giovanni Defazio

Pages: 877-885

Published online: 29 Jun 2015

AbstractforCurrent and investigated alternatives to botulinum toxin for managing blepharospasm | Full Text | References | PDF (338.5 KB)

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Molecular mechanisms of congenital hyperinsulinism and prospective therapeutic targets

Azizun Nessa (Post-Doctoral Research Associate) BSc (Hons) PhD, Sofia Asim Rahman (Post-Doctoral Research Associate) BSc (Hons) MRes PhD & Khalid Hussain (GOSHCC Professor in Paediatric Metabolic Endocrinology) MBChB MRCP MD MSc MRCPCH

Pages: 887-898

Published online: 08 Jul 2015

AbstractforMolecular mechanisms of congenital hyperinsulinism and prospective therapeutic targets | Full Text | References | PDF (659.1 KB)

90 Views
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Perspectives on gene therapy for Fanconi anemia

Susana Navarro, Paula Río & Juan A Bueren

Pages: 899-910

Published online: 13 Jul 2015

AbstractforPerspectives on gene therapy for Fanconi anemia | Full Text | References | PDF (515.1 KB)

80 Views
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Hematopoietic stem cell transplantation for metachromatic leukodystrophy

Laura Lorioli & Alessandra Biffi

Pages: 911-919

Published online: 04 Jul 2015

AbstractforHematopoietic stem cell transplantation for metachromatic leukodystrophy | Full Text | References | PDF (274.2 KB)

104 Views
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Therapeutic targets and investigated strategies for treating B-cell non-Hodgkin lymphoma

Jose C Villasboas & Stephen M Ansell

Pages: 921-932

Published online: 26 Jun 2015

AbstractforTherapeutic targets and investigated strategies for treating B-cell non-Hodgkin lymphoma | Full Text | References | PDF (740.9 KB)

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Drug Evaluation

T100: nebulized-concentrated tobramycin formulation for treatment of Pseudomonas aeruginosa infection in cystic fibrosis patients

Zarmina Ehsan & John Clancy

Pages: 933-943

Published online: 13 Jul 2015

AbstractforT100: nebulized-concentrated tobramycin formulation for treatment of Pseudomonas aeruginosa infection in cystic fibrosis patients | Full Text | References | PDF (334.8 KB)

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Nivolumab in the treatment of advanced melanoma

Kevin B Kim (Medical Director of Melanoma Clinical Research Program)

Pages: 945-956

Published online: 10 Jul 2015

AbstractforNivolumab in the treatment of advanced melanoma | Full Text | References | PDF (326.2 KB)

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Tiotropium bromide for cystic fibrosis

Saadoun Bin-Hasan MB Bch FRCP(C) & Felix Ratjen MD PhD FRCP(C) FERS

Pages: 957-966

Published online: 09 Jul 2015

AbstractforTiotropium bromide for cystic fibrosis | Full Text | References | PDF (323.6 KB)

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