Expert Opinion on Orphan Drugs, Volume 6, Issue 2 (2018)

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Expert Opinion on Orphan Drugs, Volume 6, Issue 2 (2018)

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Editorial

Editorial
Glanzmann thrombasthenia: an editorial perspective
Kanjaksha Ghosh
Pages: 91-93
Published online: 03 Jan 2018
First Page PreviewforGlanzmann thrombasthenia: an editorial perspective | Full Text | References | PDF (315.8 KB)
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Free Access

Original Research

Article
Orphan medicinal products’ access to the Bulgarian pharmaceutical market – challenges and obstacles
Maria Kamusheva, Konstantin Tachkov, Guenka Petrova, Alexandra Savova & Manoela Manova
Pages: 95-104
Published online: 28 Dec 2017
AbstractforOrphan medicinal products’ access to the Bulgarian pharmaceutical market – challenges and obstacles | Full Text | References | PDF (1.4 MB)
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Review

Review
Inherited hyperammonemias: a Contemporary view on pathogenesis and diagnosis
Evelina Maines, Giovanni Piccoli, Antonia Pascarella, Francesca Colucci & Alberto B. Burlina
Pages: 105-116
Published online: 27 Dec 2017
AbstractforInherited hyperammonemias: a Contemporary view on pathogenesis and diagnosis | Full Text | References | PDF (1.3 MB)
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Open Access
Review
Adenosine deaminase deficiency: current treatments and emerging therapeutics
Vy Hong-Diep Kim, Luis Murguia-Favela & Eyal Grunebaum
Pages: 117-125
Published online: 20 Dec 2017
AbstractforAdenosine deaminase deficiency: current treatments and emerging therapeutics | Full Text | References | PDF (761.8 KB)
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Review
Tumour necrosis factor inhibitors in enthesitis related arthritis and juvenile spondylarthropathies
Gerd Horneff
Pages: 127-140
Published online: 15 Feb 2018
AbstractforTumour necrosis factor inhibitors in enthesitis related arthritis and juvenile spondylarthropathies | Full Text | References | PDF (1.4 MB)
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Review
Advances in diagnosis and potential therapeutic options for familial chylomicronemia syndrome
Lane B. Benes, Eric J. Brandt & Michael H. Davidson
Pages: 141-149
Published online: 02 Jan 2018
AbstractforAdvances in diagnosis and potential therapeutic options for familial chylomicronemia syndrome | Full Text | References | PDF (811.8 KB)
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Drug Evaluation

Article
Rucaparib for the treatment of ovarian cancer
Sunu Lazar Cyriac, Katherine Karakasis & Amit M. Oza
Pages: 151-161
Published online: 27 Dec 2017
AbstractforRucaparib for the treatment of ovarian cancer | Full Text | References | PDF (918.1 KB)
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