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Volume 2, Issue 9

Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 2, Issue 9 (2014)

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Expert Opinion on Orphan Drugs, Volume 2, Issue 9 (2014)

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Editorial

Breaking through for breakthroughs: the problem of normal science in rare disease research

J Russell Teagarden (Senior Vice President)

Pages: 865-867

Published online: 11 Jul 2014

AbstractforBreaking through for breakthroughs: the problem of normal science in rare disease research | Full Text | References | PDF (120.7 KB)

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Free Access

Original Research

Article

Clinical trial safety population size: analysis of drug approvals for rare and common indications by FDA Center for Drug Evaluation and Research

Kathryn O’Connell MD PhD & Anne R Pariser MD

Pages: 869-875

Published online: 03 Jul 2014

AbstractforClinical trial safety population size: analysis of drug approvals for rare and common indications by FDA Center for Drug Evaluation and Research | Full Text | References | PDF (165.8 KB)

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Reviews

Review Article

Current and future therapeutic strategies to treat ataxia telangiectasia

Luciana Chessa (Professor)

Pages: 877-887

Published online: 08 Aug 2014

AbstractforCurrent and future therapeutic strategies to treat ataxia telangiectasia | Full Text | References | PDF (241.8 KB)

129 Views
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Review Article

Sézary syndrome: an overview of current and future treatment options

Sangeetha Venkatarajan (Clinical Research Fellow) MD MBA & Madeleine Duvic (Professor and Deputy Chairman) MD

Pages: 889-901

Published online: 25 Jun 2014

AbstractforSézary syndrome: an overview of current and future treatment options | Full Text | References | PDF (337.3 KB)

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Review Article

Screening, diagnosis and epidemiology of Batten disease

Natalie A Simpson, Elizabeth D Wheeler & David A Pearce

Pages: 903-910

Published online: 05 Jul 2014

AbstractforScreening, diagnosis and epidemiology of Batten disease | Full Text | References | PDF (173.6 KB)

287 Views
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Review Article

A current perspective on treatment of adrenocortical carcinoma

Massimo Terzolo MD, Silvia De Francia PhD, Arianna Ardito MD, Paola Perotti PhD, Laura Ferrari MD & Alfredo Berruti MD

Pages: 911-921

Published online: 05 Jul 2014

AbstractforA current perspective on treatment of adrenocortical carcinoma | Full Text | References | PDF (231.7 KB)

54 Views
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Review Article

Japanese encephalitis: epidemiology, prevention and current status of antiviral drug development

Tomohiro Ishikawa & Eiji Konishi

Pages: 923-936

Published online: 27 Jun 2014

AbstractforJapanese encephalitis: epidemiology, prevention and current status of antiviral drug development | Full Text | References | PDF (495.9 KB)

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Review Article

Phosphodiesterase type 5 inhibitors for stuttering priapism: recent advances

Steven D Jones Jr., Ronny BW Tan MBBS MRCSEd M Med (Surgery) FAMS(Urology) & Wayne JG Hellstrom MD FACS

Pages: 937-946

Published online: 25 Aug 2014

AbstractforPhosphodiesterase type 5 inhibitors for stuttering priapism: recent advances | Full Text | References | PDF (286.3 KB)

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Review Article

Management of Schnitzler’s syndrome

Dan Lipsker (Professor) MD PhD & Cédric Lenormand MD PhD

Pages: 947-955

Published online: 25 Aug 2014

AbstractforManagement of Schnitzler’s syndrome | Full Text | References | PDF (308.9 KB)

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Orphan Drug Designation

Article

Fresh from the designation pipeline: orphan drugs recently designated in the EU (March–April 2014)

Sabina Antoniu (Lecturer) MD PhD

Pages: 957-960

Published online: 25 Aug 2014

AbstractforFresh from the designation pipeline: orphan drugs recently designated in the EU (March–April 2014) | Full Text | References | PDF (133.6 KB)

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Drug Evaluations

Tamibarotene for the treatment of acute promyelocytic leukemia

Akihiro Takeshita, Katsuji Shinagawa, Miwa Adachi, Takaaki Ono, Toru Kiguchi & Tomoki Naoe

Pages: 961-969

Published online: 25 Aug 2014

AbstractforTamibarotene for the treatment of acute promyelocytic leukemia | Full Text | References | PDF (363 KB)

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The role of rabbit antithymocyte globulin in renal transplantation

Lyndsey J Bowman (Clinical Pharmacist) PharmD, Angelina Edwards (Transplant Nephrology Fellow) MD & Daniel C Brennan (Professor of Medicine) MD FACP

Pages: 971-987

Published online: 08 Aug 2014

AbstractforThe role of rabbit antithymocyte globulin in renal transplantation | Full Text | References | PDF (310.4 KB)

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Research Article

Article

Page: i

Published online: 25 Aug 2014

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