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Volume 8, Issue 4

Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 8, Issue 4 (2020)

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Expert Opinion on Orphan Drugs, Volume 8, Issue 4 (2020)

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Review

Druggable targets, clinical trial design and proposed pharmacological management in fibrodysplasia ossificans progressiva

Robert J. Pignolo & Frederick S. Kaplan

Pages: 101-109

Published online: 13 Apr 2020

AbstractforDruggable targets, clinical trial design and proposed pharmacological management in fibrodysplasia ossificans progressiva | Full Text | References | PDF (1.3 MB) | EPUB

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Review

Balamuthia mandrillaris: pathogenesis, diagnosis, and treatment

Mohammad Ridwane Mungroo, Naveed Ahmed Khan & Ruqaiyyah Siddiqui

Pages: 111-119

Published online: 05 May 2020

AbstractforBalamuthia mandrillaris: pathogenesis, diagnosis, and treatment | Full Text | References | PDF (999.5 KB) | EPUB

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Review

Fenfluramine hydrochloride for the treatment of Dravet syndrome

Blandine Dozières-Puyravel & Stéphane Auvin

Pages: 121-126

Published online: 26 Apr 2020

AbstractforFenfluramine hydrochloride for the treatment of Dravet syndrome | Full Text | References | PDF (781.3 KB) | EPUB

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Review

Advances in therapeutic treatment options for ANCA-associated vasculitis

Shealynn Carpenter, Jan Willem Cohen Tervaert & Elaine Yacyshyn

Pages: 127-136

Published online: 06 May 2020

AbstractforAdvances in therapeutic treatment options for ANCA-associated vasculitis | Full Text | References | PDF (826 KB) | EPUB

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Expert Opinion on Orphan Drugs (Vol.8, Iss.4, 2020)

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