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Volume 2, Issue 5

Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 2, Issue 5 (2014)

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Expert Opinion on Orphan Drugs, Volume 2, Issue 5 (2014)

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Reviews

Review Article

Clinical research for rare cancers: is it a reality in the global regulatory landscape? The international rare cancer initiative

Anastassia Negrouk (Head, Chair) MSc, Denis Lacombe (Director) MD MSc, Edward L Trimble (Director) MD MPH & Matthew Seymour (Professor, Director) MD

Pages: 433-440

Published online: 17 Feb 2014

AbstractforClinical research for rare cancers: is it a reality in the global regulatory landscape? The international rare cancer initiative | Full Text | References | PDF (233.5 KB)

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Review Article

Management and orphan drug development for acute myeloid leukemia

Haojian Zhang PhD, Shaoguang Li & Huawei Li

Pages: 441-451

Published online: 22 Feb 2014

AbstractforManagement and orphan drug development for acute myeloid leukemia | Full Text | References | PDF (273.5 KB)

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Review Article

Richter's syndrome – update on biology and management

Preetesh Jain MD DM PhD, Michael Keating MD & Susan O'Brien (Ashbel Smith Professor) MD

Pages: 453-463

Published online: 01 Mar 2014

AbstractforRichter's syndrome – update on biology and management | Full Text | References | PDF (398.2 KB)

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Review Article

Current and investigational treatments for spinal muscular atrophy

Janice Kal Van Tam PhD, Evangelia Karyka BSc & Mimoun Azzouz PhD

Pages: 465-476

Published online: 26 Mar 2014

AbstractforCurrent and investigational treatments for spinal muscular atrophy | Full Text | References | PDF (380.3 KB)

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Review Article

Practical therapeutics for skin lesions of Japanese patients with discoid lupus erythematosus

Fukumi Furukawa (Vice Director, Professor and Chairman)

Pages: 477-482

Published online: 24 Mar 2014

AbstractforPractical therapeutics for skin lesions of Japanese patients with discoid lupus erythematosus | Full Text | References | PDF (238.8 KB)

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Review Article

Current and future approaches for treatment of paraneoplastic neurological syndromes with well-characterized onconeural antibodies

Adriaan HC de Jongste MD, Joost van Rosmalen PhD, Jan W Gratama MD PhD & Peter AE Sillevis Smitt MD PhD

Pages: 483-496

Published online: 03 Apr 2014

AbstractforCurrent and future approaches for treatment of paraneoplastic neurological syndromes with well-characterized onconeural antibodies | Full Text | References | PDF (492 KB)

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Drug Evaluations

Olaparib for the treatment of ovarian cancer

Stephanie Lheureux (Clinical Research Fellow) MD PhD & Amit M Oza (Director, Cancer Clinical Research Unit/Co-Director, Drug Development) BSc FRCPC MBBS MD

Pages: 497-508

Published online: 24 Mar 2014

Abstractfor Olaparib for the treatment of ovarian cancer | Full Text | References | PDF (245.7 KB)

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Droxidopa for the treatment of neurogenic orthostatic hypotension and other symptoms of neurodegenerative disorders

Santiago Perez-Lloret MD PhD, María Verónica Rey PharmD, Anne Pavy-Le Traon MD PhD & Olivier Rascol MD PhD

Pages: 509-522

Published online: 24 Mar 2014

Abstractfor Droxidopa for the treatment of neurogenic orthostatic hypotension and other symptoms of neurodegenerative disorders | Full Text | References | PDF (456.2 KB)

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A systematic review on effectiveness and safety of eliglustat for type 1 Gaucher disease

Bouwien E Smid MD & Carla EM Hollak MD PhD

Pages: 523-529

Published online: 24 Mar 2014

AbstractforA systematic review on effectiveness and safety of eliglustat for type 1 Gaucher disease | Full Text | References | PDF (242.8 KB)

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Research Article

Article

Page: i

Published online: 23 Apr 2014

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