Expert Opinion on Orphan Drugs, Volume 5, Issue 10 (2017)

Browse this journal
Sample our Bioscience journals, sign in here to start your access, Latest two full volumes FREE to you for 14 days

Expert Opinion on Orphan Drugs, Volume 5, Issue 10 (2017)

See all volumes and issues
Vol 12, 2024 Vol 11, 2023 Vol 10, 2022 Vol 9, 2021 Vol 8, 2020 Vol 7, 2019 Vol 6, 2018 Volume 5, 2017 Vol 4, 2016 Vol 3, 2015 Vol 2, 2014 Vol 1, 2013
Download citations Download PDFs Download issue

Review

Review
Shwachman-Diamond syndrome: diagnosis, pathogenesis and prognosis
Roberto Valli, Annalisa Frattini & Antonella Minelli
Pages: 753-767
Published online: 22 Aug 2017
AbstractforShwachman-Diamond syndrome: diagnosis, pathogenesis and prognosis | Full Text | References | PDF (1.5 MB)
  • 190 Views
  • 4 CrossRef citations
  • 0Altmetric
Review
Limb girdle muscular dystrophies: clinical-genetical diagnostic update and prospects for therapy
Corrado Angelini & Marina Fanin
Pages: 769-784
Published online: 23 Aug 2017
AbstractforLimb girdle muscular dystrophies: clinical-genetical diagnostic update and prospects for therapy | Full Text | References | PDF (1.3 MB)
  • 354 Views
  • 6 CrossRef citations
  • 0Altmetric
Review
Emerging treatments and personalised medicine for ciliopathies associated with cystic kidney disease
Elisa Molinari & John A. Sayer
Pages: 785-798
Published online: 31 Aug 2017
AbstractforEmerging treatments and personalised medicine for ciliopathies associated with cystic kidney disease | Full Text | References | PDF (1.2 MB)
  • 126 Views
  • 9 CrossRef citations
  • 0Altmetric
Review
Current and potential treatment options for invasive Candida infections
A. Pyrpasopoulou, E. Iosifidis & E. Roilides
Pages: 799-812
Published online: 25 Sep 2017
AbstractforCurrent and potential treatment options for invasive Candida infections | Full Text | References | PDF (1,015.4 KB)
  • 43 Views
  • 0 CrossRef citations
  • 0Altmetric
Review
Pathogenesis, diagnosis and therapeutic strategies in WHIM syndrome immunodeficiency
Lauren E. Heusinkveld, Erin Yim, Alexander Yang, Ari B. Azani, Qian Liu, Ji-Liang Gao, David H. McDermott & Philip M. Murphy
Pages: 813-825
Published online: 25 Sep 2017
AbstractforPathogenesis, diagnosis and therapeutic strategies in WHIM syndrome immunodeficiency | Full Text | References | PDF (1.2 MB)
  • 243 Views
  • 28 CrossRef citations
  • 0Altmetric
Review
Emerging treatments for pyoderma gangrenosum
Uwe Wollina
Pages: 827-832
Published online: 11 Sep 2017
AbstractforEmerging treatments for pyoderma gangrenosum | Full Text | References | PDF (467.5 KB)
  • 1108 Views
  • 3 CrossRef citations
  • 0Altmetric

Drug Evaluation

Article
Canakinumab for the treatment of TNF-receptor associated periodic syndrome (TRAPS)
Martina Soliani & Marco Cattalini
Pages: 833-838
Published online: 19 Sep 2017
AbstractforCanakinumab for the treatment of TNF-receptor associated periodic syndrome (TRAPS) | Full Text | References | PDF (476.2 KB)
  • 43 Views
  • 1 CrossRef citations
  • 0Altmetric

Explore articles

Explore the most recently published articles

Current and emerging therapies for the treatment of leishmaniasis
Shyam Sundar et al.
Review Article | Published online: 1 Apr 2024
Open Access
The Orphan Drug Act and rare cancers: a retrospective analysis of oncologic orphan drug designations and associated approvals from 1983-2022
Abraham Viju Ipe et al.
Article | Published online: 26 Mar 2024
Open Access
Orphan drugs approved in Canada: health technology assessment, price negotiation, and government formulary listing
Nigel S B. Rawson et al.
Article | Published online: 13 Feb 2024
Open Access
Overview of genetic testing in Prader-Willi syndrome
Ying Gao et al.
Review Article | Published online: 28 Sep 2023
Open Access