Expert Opinion on Orphan Drugs, Volume 5, Issue 2 (2017)

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Expert Opinion on Orphan Drugs, Volume 5, Issue 2 (2017)

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Review

Review
Aspirin responsive erythromelalgia in JAK2-thrombocythemia and incurable inherited erythrothermalgia in neuropathic Nav1.7 sodium channelopathy: from Mitchell 1878 to Michiels 2017
Jan Jacques Michiels
Pages: 111-129
Published online: 26 Dec 2016
AbstractforAspirin responsive erythromelalgia in JAK2-thrombocythemia and incurable inherited erythrothermalgia in neuropathic Nav1.7 sodium channelopathy: from Mitchell 1878 to Michiels 2017 | Full Text | References | PDF (2.3 MB)
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Review
Galactosialidosis: historic aspects and overview of investigated and emerging treatment options
Ida Annunziata & Alessandra d’Azzo
Pages: 131-141
Published online: 14 Dec 2016
AbstractforGalactosialidosis: historic aspects and overview of investigated and emerging treatment options | Full Text | References | PDF (1.7 MB)
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Review
Managing fibrolamellar hepatocellular carcinoma
Miral Sadaria Grandhi & Timothy M. Pawlik
Pages: 143-152
Published online: 14 Dec 2016
AbstractforManaging fibrolamellar hepatocellular carcinoma | Full Text | References | PDF (1 MB)
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Review
Clinical trials examining treatments for inborn errors of amino acid metabolism
Amy Goldstein & Jerry Vockley
Pages: 153-164
Published online: 27 Dec 2016
AbstractforClinical trials examining treatments for inborn errors of amino acid metabolism | Full Text | References | PDF (849.5 KB)
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Review
Challenges and new horizons in the periodic fever, aphthous stomatitis, pharyngitis and adenitis (PFAPA) syndrome
Stefano Gentileschi, Antonio Vitale, Bruno Frediani, Mauro Galeazzi, Donato Rigante & Luca Cantarini
Pages: 165-171
Published online: 13 Jan 2017
AbstractforChallenges and new horizons in the periodic fever, aphthous stomatitis, pharyngitis and adenitis (PFAPA) syndrome | Full Text | References | PDF (576.3 KB)
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Review
Pathogenesis and treatment options for hemophilic synovitis
E. Carlos Rodriguez-Merchan & Alexander D. Liddle
Pages: 173-179
Published online: 12 Jan 2017
AbstractforPathogenesis and treatment options for hemophilic synovitis | Full Text | References | PDF (762.9 KB)
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Review
Cystic fibrosis treatment: targeting the basic defect
Pedro Mondejar-Lopez, Maria Dolores Pastor-Vivero, Manuel Sanchez-Solis & Amparo Escribano
Pages: 181-192
Published online: 18 Jan 2017
AbstractforCystic fibrosis treatment: targeting the basic defect | Full Text | References | PDF (1 MB)
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Drug Evaluation

Article
Selexipag, an orally available IP receptor agonist, in the treatment of pulmonary arterial hypertension: current evidence and future prospects
Nika Skoro-Sajer & Irene Lang
Pages: 193-200
Published online: 02 Jan 2017
AbstractforSelexipag, an orally available IP receptor agonist, in the treatment of pulmonary arterial hypertension: current evidence and future prospects | Full Text | References | PDF (740.8 KB)
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Article
Adalimumab for the treatment of non-infectious uveitis: an updated review
Buraa Kubaisi, Sarah Syeda, Alexander Schmidt & C. Stephen Foster
Pages: 201-206
Published online: 18 Jan 2017
AbstractforAdalimumab for the treatment of non-infectious uveitis: an updated review | Full Text | References | PDF (501.4 KB)
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Corrigendum

Correction
Corrigendum
Page: i
Published online: 12 Jan 2017
CitationforCorrigendum | Full Text | PDF (83.9 KB)
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Erratum

Correction
Erratum
Page: ii
Published online: 19 Jan 2017
CitationforErratum | Full Text | PDF (85.1 KB)
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