Orphan Drugs: Research and Reviews, Volume 4, Issue (2014)

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Orphan Drugs: Research and Reviews, Volume 4, Issue (2014)

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Review

Review Article
Post-marketing access to orphan drugs: a critical analysis of health technology assessment and reimbursement decision-making considerations
Georgi Iskrov & Rumen Stefanov
Pages: 1-9
Published online: 09 Jan 2014
AbstractforPost-marketing access to orphan drugs: a critical analysis of health technology assessment and reimbursement decision-making considerations | Full Text | References | PDF (216.1 KB)
  • 198 Views
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Review Article
Profile of aminopyridines for Lambert–Eaton myasthenic syndrome
Paul Maddison, Michael J Keogh & Saam Sedehizadeh
Pages: 11-18
Published online: 20 Jan 2014
AbstractforProfile of aminopyridines for Lambert–Eaton myasthenic syndrome | Full Text | References | PDF (509.4 KB)
  • 60 Views
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Open Access
Review Article
A 30-year retrospective: National Organization for Rare Disorders, the Orphan Drug Act, and the role of rare disease patient advocacy groups
Mary Dunkle
Pages: 19-27
Published online: 17 Feb 2014
AbstractforA 30-year retrospective: National Organization for Rare Disorders, the Orphan Drug Act, and the role of rare disease patient advocacy groups | Full Text | References | PDF (240.9 KB)
  • 173 Views
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Review Article
Stiripentol for the treatment of Dravet syndrome
Catherine Chiron
Pages: 29-38
Published online: 02 Apr 2014
AbstractforStiripentol for the treatment of Dravet syndrome | Full Text | References | PDF (519.1 KB)
  • 115 Views
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Open Access

Original Research

Article
Access to cross-border health care services for patients with rare diseases in the European Union
Lise Aagaard & Kent Kristensen
Pages: 39-45
Published online: 11 Apr 2014
AbstractforAccess to cross-border health care services for patients with rare diseases in the European Union | Full Text | References | PDF (225.5 KB)
  • 103 Views
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Review

Review Article
An update on gene therapy for the treatment of lipoprotein lipase deficiency
Andrew E Libby & Hong Wang
Pages: 47-54
Published online: 15 May 2014
AbstractforAn update on gene therapy for the treatment of lipoprotein lipase deficiency | Full Text | References | PDF (205.4 KB)
  • 139 Views
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Open Access
Review Article
Angiogenesis inhibitors in gastric cancer
Timothy McCarthy & Bert H O’Neil
Pages: 55-61
Published online: 14 Jul 2014
AbstractforAngiogenesis inhibitors in gastric cancer | Full Text | References | PDF (220 KB)
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Open Access
Review Article
Orphan drugs in development for urea cycle disorders: current perspectives
Johannes Häberle & Shawn E McCandless
Pages: 63-70
Published online: 30 Sep 2014
AbstractforOrphan drugs in development for urea cycle disorders: current perspectives | Full Text | References | PDF (331.5 KB)
  • 78 Views
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Open Access
Review Article
New treatments in development for Pseudomonas aeruginosa infections in the lungs of individuals with cystic fibrosis
Saeed S Akhand, Rebecca S Pettit, Tonia E Gardner & Gregory G Anderson
Pages: 71-81
Published online: 24 Oct 2014
AbstractforNew treatments in development for Pseudomonas aeruginosa infections in the lungs of individuals with cystic fibrosis | Full Text | References | PDF (284.4 KB)
  • 80 Views
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Open Access
Review Article
Orphan drugs in glioblastoma multiforme: a review
Ulrik Lassen, Morten Mau-Sørensen & Hans Skovgaard Poulsen
Pages: 83-91
Published online: 14 Nov 2014
AbstractforOrphan drugs in glioblastoma multiforme: a review | Full Text | References | PDF (234.2 KB)
  • 95 Views
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Review Article
Optimal management of interstitial lung disease associated with dermatomyositis/polymyositis: lessons from the Japanese experience
Kazuhiro Kurasawa & Satoko Arai
Pages: 93-107
Published online: 21 Nov 2014
AbstractforOptimal management of interstitial lung disease associated with dermatomyositis/polymyositis: lessons from the Japanese experience | Full Text | References | PDF (1.1 MB)
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Open Access

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