Expert Opinion on Orphan Drugs, Volume 5, Issue 4 (2017)

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Expert Opinion on Orphan Drugs, Volume 5, Issue 4 (2017)

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Editorial

Editorial
Hard targets for a second skeleton: therapeutic horizons for fibrodysplasia ossificans progressiva (FOP)
Frederick S. Kaplan, Robert J. Pignolo, Mona M. Al Mukaddam & Eileen M. Shore
Pages: 291-294
Published online: 17 Mar 2017
First Page PreviewforHard targets for a second skeleton: therapeutic horizons for fibrodysplasia ossificans progressiva (FOP) | Full Text | References | PDF (452.1 KB)
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Free Access

Review

Review
Presentation and treatments for Mucopolysaccharidosis Type II (MPS II; Hunter Syndrome)
Molly Stapleton, Francyne Kubaski, Robert W. Mason, Hiromasa Yabe, Yasuyuki Suzuki, Kenji E. Orii, Tadao Orii & Shunji Tomatsu
Pages: 295-307
Published online: 08 Mar 2017
AbstractforPresentation and treatments for Mucopolysaccharidosis Type II (MPS II; Hunter Syndrome) | Full Text | References | PDF (1.4 MB) | Supplemental
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Review
Treatment options in primary hyperoxaluria Type I
Barbara Cellini
Pages: 309-319
Published online: 28 Feb 2017
AbstractforTreatment options in primary hyperoxaluria Type I | Full Text | References | PDF (1.1 MB)
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Review
Birdshot retinochoroidopathy: pathophysiology, diagnosis and treatment
Karen Small, Mariantonia Ferrara, Alexander Schmidt & C. Stephen Foster
Pages: 321-329
Published online: 10 Mar 2017
AbstractforBirdshot retinochoroidopathy: pathophysiology, diagnosis and treatment | Full Text | References | PDF (1.1 MB)
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Review
The challenge of very rare childhood cancers in developed and developing countries
Andrea Ferrari, Dominik T. Schneider, Gianni Bisogno, Daniel Orbach, Milena Villarroel, Veronica Giron, Carlos Rodriguez-Galindo, Silvia Sorbara, Chiara Magni, Stefano Chiaravalli, Michela Casanova, Giovanni Cecchetto, Jan Godzinski, Ewa Bien, Teresa Stachowicz-Stencel, Bernadette Brennan, Yves Reguerre, Iyad Sultan & Ines B. Brecht
Pages: 331-341
Published online: 28 Feb 2017
AbstractforThe challenge of very rare childhood cancers in developed and developing countries | Full Text | References | PDF (790.4 KB)
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Review
Current and emerging treatment options for vernal keratoconjunctivitis
Marta Sacchetti, Alice Bruscolini, Irene Abicca, Marcella Nebbioso, Maurizio La Cava, Stefano Bonini & Alessandro Lambiase
Pages: 343-353
Published online: 07 Mar 2017
AbstractforCurrent and emerging treatment options for vernal keratoconjunctivitis | Full Text | References | PDF (1.2 MB)
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Review
Emerging and investigational therapies for neuroblastoma
Mark A. Applebaum, Ami V. Desai, Julia L. Glade Bender & Susan L. Cohn
Pages: 355-368
Published online: 17 Mar 2017
AbstractforEmerging and investigational therapies for neuroblastoma | Full Text | References | PDF (1.2 MB)
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Drug Evaluation

Article
Liposomal cytarabine and daunorubicin (CPX-351) for treatment of acute myeloid leukemia
Aaron Ronson, Ariella Tvito & Jacob M. Rowe
Pages: 369-374
Published online: 13 Jan 2017
AbstractforLiposomal cytarabine and daunorubicin (CPX-351) for treatment of acute myeloid leukemia | Full Text | References | PDF (648.5 KB)
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Article
Eculizumab for the treatment of hemolytic paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome and refractory myasthenia gravis
Jörg Schubert & Jan Menne
Pages: 375-379
Published online: 24 Mar 2017
AbstractforEculizumab for the treatment of hemolytic paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome and refractory myasthenia gravis | Full Text | References | PDF (402.5 KB)
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