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Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 1, Issue 4 (2013)

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Expert Opinion on Orphan Drugs, Volume 1, Issue 4 (2013)

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Editorial

Orphan drug designation – Europe, the USA and Japan

Daniel J O'Connor (Medical Assessor and UK representative on COMP) BSc MB ChB MSc PhD MFPM

Pages: 255-259

Published online: 13 Feb 2013

AbstractforOrphan drug designation – Europe, the USA and Japan | Full Text | References | PDF (175.6 KB)

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Reviews

Review Article

Treatment of eosinophilic esophagitis

Jonathan M Spergel MD PhD & Antonella Cianferoni MD PhD

Pages: 261-272

Published online: 15 Feb 2013

AbstractforTreatment of eosinophilic esophagitis | Full Text | References | PDF (368.5 KB)

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Review Article

The Australian process for subsidised access to orphan drugs for rare inherited disorders of metabolism

Jack Goldblatt (Memorial Clinical Professor, Director of Genetic Services and Familial Cancer Programme of Western Australia) AM MB ChB MD FRACP

Pages: 273-277

Published online: 02 Mar 2013

AbstractforThe Australian process for subsidised access to orphan drugs for rare inherited disorders of metabolism | Full Text | References | PDF (171.8 KB)

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Review Article

Management of epidermolysis bullosa

Mae N Ramirez MD, Dedee F Murrell MD & Jo-David Fine MD MPH FRCP

Pages: 279-293

Published online: 25 Feb 2013

AbstractforManagement of epidermolysis bullosa | Full Text | References | PDF (262 KB)

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Review Article

Contemporary management of pemphigus

Christoph M Hammers MD, Luisa Lunardon MD, Enno Schmidt MD PhD & Detlef Zillikens MD

Pages: 295-314

Published online: 06 Mar 2013

AbstractforContemporary management of pemphigus | Full Text | References | PDF (376.6 KB)

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Review Article

Females and children with Anderson–Fabry disease: diagnosis, monitoring, benefits of enzyme replacement therapy (ERT) and considerations on timing of starting ERT

Rossella Parini MD & Sandro Feriozzi MD

Pages: 315-330

Published online: 06 Mar 2013

AbstractforFemales and children with Anderson–Fabry disease: diagnosis, monitoring, benefits of enzyme replacement therapy (ERT) and considerations on timing of starting ERT | Full Text | References | PDF (445.8 KB)

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Drug Evaluations

Idebenone treatment in Leber's hereditary optic neuropathy: rationale and efficacy

Nuri Gueven PhD & Dharmesh Faldu MPharm

Pages: 331-339

Published online: 20 Feb 2013

Abstractfor Idebenone treatment in Leber's hereditary optic neuropathy: rationale and efficacy | Full Text | References | PDF (257.3 KB)

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Afamelanotide for the treatment of erythropoietic protoporphyria

Elisabeth Irene Minder

Pages: 341-349

Published online: 11 Mar 2013

Abstractfor Afamelanotide for the treatment of erythropoietic protoporphyria | Full Text | References | PDF (387.7 KB)

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Research Article

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Published online: 11 Mar 2013

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