Expert Opinion on Orphan Drugs, Volume 5, Issue 12 (2017)

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Expert Opinion on Orphan Drugs, Volume 5, Issue 12 (2017)

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Original Research

Article
Patient access to orphan drugs
Joshua P. Cohen & Josephine G. Awatin
Pages: 923-932
Published online: 20 Nov 2017
AbstractforPatient access to orphan drugs | Full Text | References | PDF (742.7 KB)
  • 861 Views
  • 6 CrossRef citations
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Review

Review
Recognition, diagnosis and treatment of homozygous familial hypercholesterolemia
Ezim Ajufo & Marina Cuchel
Pages: 933-943
Published online: 05 Nov 2017
AbstractforRecognition, diagnosis and treatment of homozygous familial hypercholesterolemia | Full Text | References | PDF (1.1 MB)
  • 56 Views
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Review
Mucosal melanoma: epidemiology, biology, management and the role of immunotherapy
Sherry Shen, Jessica Yang & Richard D. Carvajal
Pages: 945-952
Published online: 06 Nov 2017
AbstractforMucosal melanoma: epidemiology, biology, management and the role of immunotherapy | Full Text | References | PDF (602.3 KB)
  • 71 Views
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Review
Addressing the challenges of lymphangioleiomyomatosis assessment in the clinic
Angelo M. Taveira-DaSilva & Joel Moss
Pages: 953-965
Published online: 15 Nov 2017
AbstractforAddressing the challenges of lymphangioleiomyomatosis assessment in the clinic | Full Text | References | PDF (1.7 MB)
  • 22 Views
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Review
Advances in the genetic understanding of hypohidrotic ectodermal dysplasia
Toshihide Higashino, John Y. W. Lee & John A. McGrath
Pages: 967-975
Published online: 26 Nov 2017
AbstractforAdvances in the genetic understanding of hypohidrotic ectodermal dysplasia | Full Text | References | PDF (767 KB)
  • 78 Views
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Review
Pharmacological approaches for treating glycogen storage disorders involving polyglucosan body accumulation
Or Kakhlon
Pages: 977-982
Published online: 20 Nov 2017
AbstractforPharmacological approaches for treating glycogen storage disorders involving polyglucosan body accumulation | Full Text | References | PDF (569.1 KB)
  • 56 Views
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Review
The potential of emerging therapeutics for epithelioid sarcoma
Brittany L. Siontis, Rashmi Chugh & Scott M. Schuetze
Pages: 983-989
Published online: 22 Nov 2017
AbstractforThe potential of emerging therapeutics for epithelioid sarcoma | Full Text | References | PDF (835.9 KB)
  • 67 Views
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Drug Evaluation

Article
Ceritinib: an orphan drug for ALK positive non-small cell lung cancer with robust clinical evidence
Christian Rolfo, David Arias, Jose Ferri Gandia, Paolo Manca, Edgardo Santos & Luis E. Raez
Pages: 991-997
Published online: 24 Nov 2017
AbstractforCeritinib: an orphan drug for ALK positive non-small cell lung cancer with robust clinical evidence | Full Text | References | PDF (843.5 KB)
  • 59 Views
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Article
Glycerol phenylbutyrate for the maintenance treatment of patients with deficiencies in enzymes of the urea cycle
Nicola Longo & Robert J. Holt
Pages: 999-1010
Published online: 28 Nov 2017
AbstractforGlycerol phenylbutyrate for the maintenance treatment of patients with deficiencies in enzymes of the urea cycle | Full Text | References | PDF (1.4 MB)
  • 3765 Views
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