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Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 3, Issue 4 (2015)

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Expert Opinion on Orphan Drugs, Volume 3, Issue 4 (2015)

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Editorial

What the future holds for the genetic diagnosis for neurodegeneration with brain iron accumulation syndromes?

Susanne A Schneider & Manju A Kurian

Pages: 353-356

Published online: 26 Mar 2015

AbstractforWhat the future holds for the genetic diagnosis for neurodegeneration with brain iron accumulation syndromes? | Full Text | References | PDF (137.6 KB)

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Review

Neonatal hypoxic ischaemic encephalopathy: current and future treatment options

Nazakat M Merchant MBBS MRCPCH, Denis V Azzopardi & Anthony David Edwards

Pages: 357-377

Published online: 24 Mar 2015

AbstractforNeonatal hypoxic ischaemic encephalopathy: current and future treatment options | Full Text | References | PDF (887.1 KB)

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Genetics of the Ehlers–Danlos syndrome: more than collagen disorders

Tim Van Damme MD, Delfien Syx MSc, Paul Coucke PhD, Sofie Symoens PhD, Anne De Paepe MD PhD & Fransiska Malfait MD PhD

Pages: 379-392

Published online: 07 Mar 2015

AbstractforGenetics of the Ehlers–Danlos syndrome: more than collagen disorders | Full Text | References | PDF (525.4 KB)

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The importance of biobank and nationwide registry for lymphangioleiomyomatosis in a small sized country

Marjolijn HL Struik MSc, Coline HM van Moorsel PhD, Frouke T van Beek MD, Marcel Veltkamp MD PhD, Francis X McCormack MD & Jan C Grutters MD PhD

Pages: 393-401

Published online: 26 Mar 2015

AbstractforThe importance of biobank and nationwide registry for lymphangioleiomyomatosis in a small sized country | Full Text | References | PDF (214.4 KB)

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Achalasia: new diagnostic tests and treatments

Joel E Richter (Professor of Medicine, Hugh F. Culverhouse Chair for Esophageal Disorders, Director) MD FACP MACG

Pages: 403-417

Published online: 02 Mar 2015

AbstractforAchalasia: new diagnostic tests and treatments | Full Text | References | PDF (791.2 KB)

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Current and future pharmacotherapy for alopecia areata

Norma Elizabeth Vázquez-Herrera & Antonella Tosti

Pages: 419-431

Published online: 06 Mar 2015

AbstractforCurrent and future pharmacotherapy for alopecia areata | Full Text | References | PDF (640.8 KB)

171 Views
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Enzyme replacement and gene therapy for mucopolysaccharidoses: current progress and future directions

Michael Beck MD

Pages: 433-444

Published online: 04 Mar 2015

AbstractforEnzyme replacement and gene therapy for mucopolysaccharidoses: current progress and future directions | Full Text | References | PDF (279.5 KB)

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Orphan drug approvals of 2014: Europe and the United States

Peter Norman MBA PhD

Pages: 445-455

Published online: 04 Mar 2015

AbstractforOrphan drug approvals of 2014: Europe and the United States | Full Text | References | PDF (237.4 KB)

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Drug Evaluation

Aldoxorubicin for the treatment of advanced soft tissue sarcoma

Kamalesh K Sankhala MD, Neal S Chawla MD & Sant P Chawla MD

Pages: 457-466

Published online: 26 Mar 2015

AbstractforAldoxorubicin for the treatment of advanced soft tissue sarcoma | Full Text | References | PDF (394.3 KB)

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Plerixafor: potential role in acute leukemia therapy

Jane Liesveld (Professor of Medicine) MD

Pages: 467-475

Published online: 14 Mar 2015

AbstractforPlerixafor: potential role in acute leukemia therapy | Full Text | References | PDF (273.7 KB)

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Blinatumomab for treating acute lymphoblastic leukemia

Josep-Maria Ribera MD PhD, Susana Vives, Cristina Motlló & María-José Jiménez-Lorenzo

Pages: 477-485

Published online: 15 Mar 2015

AbstractforBlinatumomab for treating acute lymphoblastic leukemia | Full Text | References | PDF (220.1 KB)

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Expert Opinion on Orphan Drugs (Vol.3, Iss.4, 2015)

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