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Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 2, Issue 6 (2014)

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Expert Opinion on Orphan Drugs, Volume 2, Issue 6 (2014)

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Reviews

Review Article

Targeted treatments in fragile X syndrome

Emma B Hare (Certified Clinical Research Coordinator) BA, Randi J Hagerman (Medical Director of the MIND Institute) MD & Reymundo Lozano MD

Pages: 531-543

Published online: 04 Apr 2014

AbstractforTargeted treatments in fragile X syndrome | Full Text | References | PDF (564.9 KB)

158 Views
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Review Article

An overview of current and future therapeutic strategies for the treatment of primary sclerosing cholangitis

Ahmad H Ali MBBS, Elizabeth J Carey (Assistant Professor of Medicine) MD & Keith D Lindor (Professor of Medicine, Executive Vice Provost and Dean) MD

Pages: 545-556

Published online: 19 Apr 2014

AbstractforAn overview of current and future therapeutic strategies for the treatment of primary sclerosing cholangitis | Full Text | References | PDF (288.1 KB)

119 Views
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Review Article

Chordoma: an assessment of future treatment and management strategies

Matthias Uhl MD & Jürgen Debus MD PhD

Pages: 557-565

Published online: 16 Apr 2014

AbstractforChordoma: an assessment of future treatment and management strategies | Full Text | References | PDF (250.6 KB)

93 Views
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Review Article

Pseudoxanthoma elasticum: diagnostic features, classification and treatment options

Jouni Uitto MD PhD, Qiujie Jiang MD PhD, András Váradi PhD, Lionel G Bercovitch MD & Sharon F Terry MA

Pages: 567-577

Published online: 21 Apr 2014

AbstractforPseudoxanthoma elasticum: diagnostic features, classification and treatment options | Full Text | References | PDF (400.3 KB)

148 Views
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Review Article

Treatment options for alveolar soft part sarcoma in pediatric patients

Gianni Bisogno MD PhD, Andrea Ferrari MD, Rita Alaggio MD & Daniel Orbach MD

Pages: 579-589

Published online: 22 May 2014

AbstractforTreatment options for alveolar soft part sarcoma in pediatric patients | Full Text | References | PDF (247.2 KB)

67 Views
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Review Article

Paget’s disease of bone: epidemiology, pathogenesis and pharmacotherapy

Luigi Gennari MD PhD, Daniela Merlotti MD PhD, Domenico Rendina MD, Fernando Gianfrancesco PhD, Teresa Esposito PhD & Ranuccio Nuti MD

Pages: 591-603

Published online: 22 May 2014

AbstractforPaget’s disease of bone: epidemiology, pathogenesis and pharmacotherapy | Full Text | References | PDF (614.6 KB)

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Drug Evaluations

Pazopanib for the treatment of advanced renal cell cancer

Bernard Escudier MD & Laurence Albiges MD PhD

Pages: 605-616

Published online: 09 May 2014

Abstractfor Pazopanib for the treatment of advanced renal cell cancer | Full Text | References | PDF (497.5 KB)

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Bendamustine in the treatment of chronic lymphocytic leukemia

Chaitra Ujjani MD & Bruce D Cheson MD

Pages: 617-623

Published online: 21 May 2014

Abstractfor Bendamustine in the treatment of chronic lymphocytic leukemia | Full Text | References | PDF (226.1 KB)

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Denileukin diftitox for the treatment of cutaneous T-cell lymphoma

H Miles Prince MD & Kate M Newland FACD

Pages: 625-634

Published online: 26 Apr 2014

Abstractfor Denileukin diftitox for the treatment of cutaneous T-cell lymphoma | Full Text | References | PDF (268.7 KB)

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Orphan Drug Designation

Article

Fresh from the designation pipeline: orphan drugs recently designated in the European Union (December 2013-February 2014)

Sabina Antoniu (Lecturer) MD PhD

Pages: 635-641

Published online: 19 Apr 2014

AbstractforFresh from the designation pipeline: orphan drugs recently designated in the European Union (December 2013-February 2014) | Full Text | References | PDF (164.6 KB)

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Research Article

Article

Page: i

Published online: 22 May 2014

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Expert Opinion on Orphan Drugs (Vol.2, Iss.6, 2014)

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