Expert Opinion on Orphan Drugs, Volume 5, Issue 8 (2017)

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Expert Opinion on Orphan Drugs, Volume 5, Issue 8 (2017)

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Editorial

Editorial
Ultra-orphan drugs: can we afford the price
Devidas Menon & Tania Stafinski
Pages: 611-612
Published online: 19 Jun 2017
CitationforUltra-orphan drugs: can we afford the price | Full Text | References | PDF (256.2 KB)
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Review

Review
Cultural, geographical and ethical questions when looking to enroll pediatric patients in rare disease clinical trials
Stefan Anzelewicz, Hanna Garnier, Arun Rangaswami & Piotr Czauderna
Pages: 613-621
Published online: 03 Jul 2017
AbstractforCultural, geographical and ethical questions when looking to enroll pediatric patients in rare disease clinical trials | Full Text | References | PDF (709.6 KB)
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Review
The management of neurofibromatosis type 1-associated malignant peripheral nerve sheath tumors: challenges, progress, and future prospects
Angela C. Hirbe & David H Gutmann
Pages: 623-631
Published online: 11 Jul 2017
AbstractforThe management of neurofibromatosis type 1-associated malignant peripheral nerve sheath tumors: challenges, progress, and future prospects | Full Text | References | PDF (1.1 MB)
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Review
Therapeutic outlook for Castleman’s disease: prospects for the next decade
Tomohiro Koga, Shino Fujimoto, Atsushi Kawakami, Hiroshi Kawabata, Yasufumi Masaki, Tadamitsu Kishimoto & Kazuyuki Yoshizaki
Pages: 633-640
Published online: 05 Jul 2017
AbstractforTherapeutic outlook for Castleman’s disease: prospects for the next decade | Full Text | References | PDF (1 MB)
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Review
Glanzmann’s thrombasthenia: strategies for identification and management
Man-Chiu Poon, Giovanni Di Minno, Rainer Zotz & Roseline d’Oiron
Pages: 641-653
Published online: 28 Jun 2017
AbstractforGlanzmann’s thrombasthenia: strategies for identification and management | Full Text | References | PDF (1.2 MB)
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Review
Clinical trials evaluating potential therapies for light chain (AL) amyloidosis
Eli Muchtar & Morie A. Gertz
Pages: 655-663
Published online: 19 Jun 2017
AbstractforClinical trials evaluating potential therapies for light chain (AL) amyloidosis | Full Text | References | PDF (1.2 MB)
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Review
Current and emerging pharmacotherapies for hereditary hemorrhagic telangiectasia
Virginia Albiñana, Lucia Recio-Poveda, Roberto Zarrabeitia & Luisa M. Botella
Pages: 665-675
Published online: 17 Jul 2017
AbstractforCurrent and emerging pharmacotherapies for hereditary hemorrhagic telangiectasia | Full Text | References | PDF (1.3 MB)
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Drug Evaluation

Article
Apremilast for the treatment of Behcet’s syndrome
Gulen Hatemi & Yusuf Yazici
Pages: 677-681
Published online: 24 Jul 2017
AbstractforApremilast for the treatment of Behcet’s syndrome | Full Text | References | PDF (401.8 KB)
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