Expert Opinion on Orphan Drugs, Volume 6, Issue 8 (2018)

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Expert Opinion on Orphan Drugs, Volume 6, Issue 8 (2018)

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Drug Evaluation

Article
Ramucirumab for the treatment of gastric adenocarcinoma
Maria Vassilakopoulou, Kazuto Harada & Jaffer A Ajani
Pages: 449-455
Published online: 20 Jul 2018
AbstractforRamucirumab for the treatment of gastric adenocarcinoma | Full Text | References | PDF (836.9 KB)
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Article
Voretigene neparvovec-rzyl for the treatment of biallelic RPE65 mutation–associated retinal dystrophy
Stephen Russell, Jean Bennett, Albert M. Maguire & Katherine A. High
Pages: 457-464
Published online: 16 Aug 2018
AbstractforVoretigene neparvovec-rzyl for the treatment of biallelic RPE65 mutation–associated retinal dystrophy | Full Text | References | PDF (1.1 MB)
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Review

Review
The role of cancer stem cells and the therapeutic potential of TRX-E-002-1 in ovarian cancer
Muhammad Saif, E. I. Ager, Penelope Field & K. J. Lilischkis
Pages: 465-475
Published online: 28 Aug 2018
AbstractforThe role of cancer stem cells and the therapeutic potential of TRX-E-002-1 in ovarian cancer | Full Text | References | PDF (1,023.8 KB)
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Review
Hemophilic arthropathy: current treatment challenges and future prospects
E. Carlos Rodriguez-Merchan
Pages: 477-483
Published online: 30 Aug 2018
AbstractforHemophilic arthropathy: current treatment challenges and future prospects | Full Text | References | PDF (905 KB)
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Review
Paget’s disease of bone: an update on epidemiology, pathogenesis and pharmacotherapy
Luigi Gennari, Domenico Rendina, Tommaso Picchioni, Simone Bianciardi, Maria Materozzi, Ranuccio Nuti & Daniela Merlotti
Pages: 485-496
Published online: 23 Jul 2018
AbstractforPaget’s disease of bone: an update on epidemiology, pathogenesis and pharmacotherapy | Full Text | References | PDF (1.1 MB)
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Original Research

Article
Evaluating the impact of peer support and connection on the quality of life of patients with familial chylomicronemia syndrome
Valerie Salvatore, Alan Gilstrap, Karren R Williams, Swati Thorat, Michael Stevenson, Andrea R Gwosdow, Andrew Hsieh, Brant C Hubbard & David Davidson
Pages: 497-505
Published online: 05 Aug 2018
AbstractforEvaluating the impact of peer support and connection on the quality of life of patients with familial chylomicronemia syndrome | Full Text | References | PDF (1.6 MB) | Supplemental
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