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Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 3, Issue 1 (2015)

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Expert Opinion on Orphan Drugs, Volume 3, Issue 1 (2015)

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Editorial

Dormant therapies: hope for the rare disease community

D Wayne Taylor (Executive Director) PhD FCIM

Pages: 1-3

Published online: 25 Nov 2014

AbstractforDormant therapies: hope for the rare disease community | Full Text | References | PDF (121.7 KB)

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Review

α-1 antitrypsin deficiency: current and future treatment options

Oliver J McElvaney, Abdelhaleem M E Bella & Noel G McElvaney (Professor of Medicine)

Pages: 5-14

Published online: 23 Dec 2014

Abstractforα-1 antitrypsin deficiency: current and future treatment options | Full Text | References | PDF (253.1 KB)

126 Views
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Clinical features, genetics and potential therapeutic approaches for Birt–Hogg–Dubé syndrome

Laura S Schmidt & W Marston Linehan MD

Pages: 15-29

Published online: 29 Nov 2014

AbstractforClinical features, genetics and potential therapeutic approaches for Birt–Hogg–Dubé syndrome | Full Text | References | PDF (637.2 KB)

196 Views
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Genetics, diagnosis and future treatment strategies for primary ciliary dyskinesia

Mary Leigh Anne Daniels MD MPH & Peadar G Noone MD FCCP FRCPI

Pages: 31-44

Published online: 29 Nov 2014

AbstractforGenetics, diagnosis and future treatment strategies for primary ciliary dyskinesia | Full Text | References | PDF (508.2 KB)

132 Views
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Therapeutic strategies for Cushing’s syndrome: an update

Filippo Ceccato (Endocrinologist) MD, Mattia Barbot (Endocrinologist) MD, Marialuisa Zilio (Endocrinologist) MD, Nora Albiger (Endocrinologist) MD, Franco Mantero (Endocrinologist) MD & Carla Scaroni (Endocrinologist) MD

Pages: 45-56

Published online: 10 Dec 2014

AbstractforTherapeutic strategies for Cushing’s syndrome: an update | Full Text | References | PDF (333 KB)

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The future for treating Creutzfeldt–Jakob disease

Vito Vetrugno PhD, Maria Puopolo DStat, Franco Cardone PhD, Fiorentino Capozzoli MD, Anna Ladogana MD & Maurizio Pocchiari MD

Pages: 57-74

Published online: 23 Dec 2014

AbstractforThe future for treating Creutzfeldt–Jakob disease | Full Text | References | PDF (839.9 KB)

278 Views
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Pharmacotherapy of placental site and epithelioid trophoblastic tumours

Fiona Taylor & Barry W Hancock

Pages: 75-85

Published online: 23 Dec 2014

AbstractforPharmacotherapy of placental site and epithelioid trophoblastic tumours | Full Text | References | PDF (412.3 KB) | Supplemental

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Drug Evaluation

Beclomethasone dipropionate for the treatment of ulcerative colitis

Tommaso Gabbani MD, Natalia Manetti MD, Siro Bagnoli MD & Vito Annese MD

Pages: 87-96

Published online: 26 Nov 2014

AbstractforBeclomethasone dipropionate for the treatment of ulcerative colitis | Full Text | References | PDF (248.1 KB)

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Efficacy and tolerability of treatment with pegvisomant in acromegaly: an overview of literature

Lucia Ferreri MD, Renata S Auriemma MD, Ludovica FS Grasso MD, Rosario Pivonello (Assistant Professor) MD PhD & Annamaria Colao (Professor) MD PhD

Pages: 97-108

Published online: 29 Oct 2014

AbstractforEfficacy and tolerability of treatment with pegvisomant in acromegaly: an overview of literature | Full Text | References | PDF (292 KB)

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Idelalisib for the treatment of B-cell malignancies

Qingshan Yang PhD, Prexy Modi, Srinivasan Ramanathan PhD, Christophe Quéva PhD & Varsha Gandhi PhD

Pages: 109-123

Published online: 05 Nov 2014

AbstractforIdelalisib for the treatment of B-cell malignancies | Full Text | References | PDF (609.9 KB)

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