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Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 1, Issue 2 (2013)

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Expert Opinion on Orphan Drugs, Volume 1, Issue 2 (2013)

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Research Article

Article

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Page: 1

Published online: 22 Jan 2013

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Editorial

Funding Grants for Rare Diseases at the Food and Drug Administration

Soumya Patel (Pharmacist Reviewer) PharmD & Katherine I Miller Needleman (Director, Orphan Products Grants Program) MS PhD RAC

Pages: 99-102

Published online: 22 Jan 2013

AbstractforFunding Grants for Rare Diseases at the Food and Drug Administration | Full Text | References | PDF (164.8 KB)

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Free Access

Reviews

Review Article

Recent advances in the treatment of adult-onset Still's disease

Der-Yuan Chen

Pages: 103-113

Published online: 21 Dec 2012

AbstractforRecent advances in the treatment of adult-onset Still's disease | Full Text | References | PDF (294.4 KB)

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Review Article

Inherited pediatric metabolic epilepsies

Phillip L Pearl MD & Yuezhou Yu

Pages: 115-129

Published online: 08 Jan 2013

AbstractforInherited pediatric metabolic epilepsies | Full Text | References | PDF (760.6 KB)

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Review Article

Orphan drug approvals in Europe since 2001: an analysis by indication

Peter Norman MBA PhD

Pages: 131-139

Published online: 08 Jan 2013

AbstractforOrphan drug approvals in Europe since 2001: an analysis by indication | Full Text | References | PDF (506.5 KB)

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Review Article

Orphan drugs for the treatment of hereditary angioedema

Henriette Farkas MD PhD DSc

Pages: 141-156

Published online: 07 Jan 2013

AbstractforOrphan drugs for the treatment of hereditary angioedema | Full Text | References | PDF (448.9 KB)

81 Views
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Review Article

Treatment of common variable immune deficiency

Elena S Resnick MD & Charlotte Cunningham-Rundles MD PhD

Pages: 157-166

Published online: 22 Jan 2013

AbstractforTreatment of common variable immune deficiency | Full Text | References | PDF (439.1 KB)

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Drug Evaluations

Article

Eculizumab treatment of atypical hemolytic uremic syndrome

Bernard S Kaplan, Rebecca L Ruebner & Lawrence Copelovitch

Pages: 167-176

Published online: 22 Jan 2013

AbstractforEculizumab treatment of atypical hemolytic uremic syndrome | Full Text | References | PDF (218.1 KB)

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Article

Oxabact: truly a new treatment option in patients with (primary) hyperoxaluria?

Bernd Hoppe

Pages: 177-184

Published online: 22 Jan 2013

Abstractfor Oxabact: truly a new treatment option in patients with (primary) hyperoxaluria? | Full Text | References | PDF (323.6 KB)

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