Expert Opinion on Orphan Drugs, Volume 5, Issue 7 (2017)

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Expert Opinion on Orphan Drugs, Volume 5, Issue 7 (2017)

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Review

Review
New insights in primary ciliary dyskinesia
Ana Reula, JS Lucas, Antonio Moreno-Galdó, Teresa Romero, Xavier Milara, Carmen Carda, Manuel Mata-Roig, Amparo Escribano, Francisco Dasi & Miguel Armengot-Carceller
Pages: 537-548
Published online: 15 May 2017
AbstractforNew insights in primary ciliary dyskinesia | Full Text | References | PDF (1 MB)
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  • 11 CrossRef citations
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Review
Advances in therapeutic options for esophageal and esophagogastric junctional adenocarcinoma
Dilsa Mizrak Kaya, Kazuto Harada & Jaffer A. Ajani
Pages: 549-557
Published online: 22 May 2017
AbstractforAdvances in therapeutic options for esophageal and esophagogastric junctional adenocarcinoma | Full Text | References | PDF (796.5 KB)
  • 81 Views
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Review
Agammaglobulinemia: comorbidities and long-term therapeutic risks
Farzaneh Rahmani, Asghar Aghamohammadi, Hans D. Ochs & Nima Rezaei
Pages: 559-574
Published online: 24 May 2017
AbstractforAgammaglobulinemia: comorbidities and long-term therapeutic risks | Full Text | References | PDF (1.1 MB)
  • 77 Views
  • 1 CrossRef citations
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Review
Therapeutic options for Progressive Supranuclear Palsy including investigational drugs
Ali Shoeibi & Irene Litvan
Pages: 575-587
Published online: 12 Jun 2017
AbstractforTherapeutic options for Progressive Supranuclear Palsy including investigational drugs | Full Text | References | PDF (1.3 MB)
  • 127 Views
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Review
Current and future approaches to overcoming the challenges of hemophilia treatment personalization
Alessandro Gringeri, Katharina Steinitz-Trost & Jennifer Doralt
Pages: 589-597
Published online: 01 Jun 2017
AbstractforCurrent and future approaches to overcoming the challenges of hemophilia treatment personalization | Full Text | References | PDF (670.5 KB)
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Review
Apert syndrome: the Paris and Rotterdam philosophy
C. Driessen, M. L. C. van Veelen, K. F. M. Joosten, S. L. Versnel, C. A. van Nieuwenhoven, E. B. Wolvius, H. H. Bredero-Boelhouwer, E. Arnaud & I. M. J. Mathijssen
Pages: 599-605
Published online: 05 Jun 2017
AbstractforApert syndrome: the Paris and Rotterdam philosophy | Full Text | References | PDF (796.5 KB)
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Open Access

Drug Evaluation

Article
Brachyury-targeting immunotherapy for chordomas: treatment with GI-6301, a Saccharomyces cerevisiae yeast-based cancer vaccine
Alia Hdeib & James M. Wright
Pages: 607-610
Published online: 05 Jun 2017
AbstractforBrachyury-targeting immunotherapy for chordomas: treatment with GI-6301, a Saccharomyces cerevisiae yeast-based cancer vaccine | Full Text | References | PDF (389 KB)
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