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Volume 4, Issue 11

Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 4, Issue 11 (2016)

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Expert Opinion on Orphan Drugs, Volume 4, Issue 11 (2016)

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Editorial

Applying integrated care systems to rare diseases

Réjean Hébert

Pages: 1095-1097

Published online: 10 Sep 2016

First Page PreviewforApplying integrated care systems to rare diseases | Full Text | References | PDF (248.6 KB)

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Drug Evaluation

Article

Antiangiogenic virotherapy: VB-111 targeting glioma

Simon Khagi & Katherine B. Peters

Pages: 1099-1103

Published online: 26 Sep 2016

AbstractforAntiangiogenic virotherapy: VB-111 targeting glioma | Full Text | References | PDF (447 KB)

358 Views
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Article

The combination of vemurafenib and cobimetinib in advanced melanoma

Lavinia Spain, Emily Goode, Yevette McGovern, Kroopa Joshi & James Larkin

Pages: 1105-1111

Published online: 11 Oct 2016

AbstractforThe combination of vemurafenib and cobimetinib in advanced melanoma | Full Text | References | PDF (636.3 KB)

118 Views
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Review

Tissue chips to aid drug development and modeling for rare diseases

Lucie A. Low & Danilo A. Tagle

Pages: 1113-1121

Published online: 19 Oct 2016

AbstractforTissue chips to aid drug development and modeling for rare diseases | Full Text | References | PDF (878.7 KB)

205 Views
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Review

Brain biomarkers and neuroimaging to diagnose urea cycle disorders and assess prognosis

Emil Barkovich, Chelsea Robinson & Andrea Gropman

Pages: 1123-1132

Published online: 08 Oct 2016

AbstractforBrain biomarkers and neuroimaging to diagnose urea cycle disorders and assess prognosis | Full Text | References | PDF (1.2 MB)

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Review

Managing of giant cell arteritis and polymyalgia rheumatica

Miguel A. González-Gay & Santos Castañeda

Pages: 1133-1144

Published online: 11 Oct 2016

AbstractforManaging of giant cell arteritis and polymyalgia rheumatica | Full Text | References | PDF (892.9 KB)

64 Views
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Review

Pain in Ehlers-Danlos syndromes: manifestations, therapeutic strategies and future perspectives

Marco Castori

Pages: 1145-1158

Published online: 27 Sep 2016

AbstractforPain in Ehlers-Danlos syndromes: manifestations, therapeutic strategies and future perspectives | Full Text | References | PDF (1.5 MB)

387 Views
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Review

Managing dystrophinopathic cardiomyopathy

Luisa Politano & Giovanni Nigro

Pages: 1159-1178

Published online: 20 Sep 2016

AbstractforManaging dystrophinopathic cardiomyopathy | Full Text | References | PDF (2.3 MB)

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Review

Pharmacological therapeutics targeting the secondary defects and downstream pathology of Duchenne muscular dystrophy

Janelle M. Spinazzola & Louis M. Kunkel

Pages: 1179-1194

Published online: 18 Oct 2016

AbstractforPharmacological therapeutics targeting the secondary defects and downstream pathology of Duchenne muscular dystrophy | Full Text | References | PDF (1.3 MB)

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