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Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 1, Issue 7 (2013)

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Expert Opinion on Orphan Drugs, Volume 1, Issue 7 (2013)

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Editorials

Editorial

Expedited programs for drug development and approval

Anne R Pariser (Associate Director for Rare Diseases) MD, Melissa Robb & Rachel E Sherman (Associate Director for Medical Policy) MD MPH

Pages: 507-510

Published online: 31 May 2013

AbstractforExpedited programs for drug development and approval | Full Text | References | PDF (226.8 KB)

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Editorial

Alternative strategies in orphan drug development

Anthony K Hall (Co-founder of Findacure) MBBS BSc AKC & Nicolas Sireau (Chairman and CEO of the AKU Society, Chairman and Co-founder of Findacure) BA (Oxon) History and Economics, MA Journalism, MSC Business Management, PhD in Social Psychology

Pages: 511-514

Published online: 22 Jun 2013

AbstractforAlternative strategies in orphan drug development | Full Text | References | PDF (219.2 KB)

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Reviews

Review Article

Short bowel syndrome – characterisation of an orphan condition with many phenotypes

Palle Bekker Jeppesen MD PhD

Pages: 515-525

Published online: 26 Jun 2013

AbstractforShort bowel syndrome – characterisation of an orphan condition with many phenotypes | Full Text | References | PDF (594.9 KB)

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Review Article

The non-surgical treatment of adult patients with short bowel syndrome

Palle Bekker Jeppesen (Associate Professor) MD PhD

Pages: 527-538

Published online: 28 Jun 2013

AbstractforThe non-surgical treatment of adult patients with short bowel syndrome | Full Text | References | PDF (351 KB)

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Review Article

Pomalidomide and the growing role of immunomodulatory agents in the treatment of myelofibrosis

Kathryn B Bollin MD, Holly L Geyer MD & Ruben A Mesa MD

Pages: 539-547

Published online: 03 Jul 2013

AbstractforPomalidomide and the growing role of immunomodulatory agents in the treatment of myelofibrosis | Full Text | References | PDF (291.4 KB)

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Review Article

Aerosolised levofloxacin in cystic fibrosis

Gerd Döring & Axel Dalhoff

Pages: 549-556

Published online: 03 Jul 2013

AbstractforAerosolised levofloxacin in cystic fibrosis | Full Text | References | PDF (308.6 KB)

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Drug Evaluations

Pasireotide for the treatment of Cushing's disease

Annamaria Colao, Chiara Simeoli, Monica De Leo, Alessia Cozzolino & Rosario Pivonello

Pages: 557-567

Published online: 17 Jun 2013

Abstractfor Pasireotide for the treatment of Cushing's disease | Full Text | References | PDF (724.8 KB)

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Plitidepsin: an orphan drug

Alina Danu, Christophe Willekens & Vincent Ribrag MD

Pages: 569-580

Published online: 19 Jun 2013

Abstractfor Plitidepsin: an orphan drug | Full Text | References | PDF (396.6 KB)

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Research Article

Article

Page: i

Published online: 03 Jul 2013

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