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Volume 4, Issue 7

Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 4, Issue 7 (2016)

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Expert Opinion on Orphan Drugs, Volume 4, Issue 7 (2016)

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Editorial

The value of collaborative research in rare disease research

Ellen J. Salkeld

Pages: 687-689

Published online: 10 May 2016

First Page PreviewforThe value of collaborative research in rare disease research | Full Text | References | PDF (292.7 KB)

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Original Research

Article

Patient participation in the development of a clinical guideline for inherited retinal dystrophies

P. Serrano-Aguilar, M. M. Trujillo-Martin, T. del Pino-Sedeño, A. Pérez de la Rosa, A. M. de Pascual y Medina, L. Perestelo-Pérez, A. Toledo-Chávarri, M. Posada de la Paz, A. Sarría Santamera & the Spanish Inherited Retinal Dystrophies CPG Development Group

Pages: 691-697

Published online: 12 May 2016

AbstractforPatient participation in the development of a clinical guideline for inherited retinal dystrophies | Full Text | References | PDF (611.1 KB) | Supplemental

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Review

Screening for genetic mutations in children and adolescents with dyslipidemia: importance of early identification and implications of missed diagnoses

Don P. Wilson, Alejandro de la Torre, Ariel Brautbar & Luke Hamilton

Pages: 699-710

Published online: 06 Jun 2016

AbstractforScreening for genetic mutations in children and adolescents with dyslipidemia: importance of early identification and implications of missed diagnoses | Full Text | References | PDF (881.3 KB)

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Review

Prospect and progress of gene therapy in acute intermittent porphyria

Delia D’Avola & Gloria Gonzalez Aseguinolaza

Pages: 711-717

Published online: 02 Jun 2016

AbstractforProspect and progress of gene therapy in acute intermittent porphyria | Full Text | References | PDF (827 KB)

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Review

Neurological manifestations in Wilson’s disease –possible treatment options for symptoms

Tomasz Litwin, Petr Dusek & Anna Czlonkowska

Pages: 719-728

Published online: 23 May 2016

AbstractforNeurological manifestations in Wilson’s disease –possible treatment options for symptoms | Full Text | References | PDF (767.1 KB)

131 Views
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Review

Overview of existing initiatives to develop and improve access and data sharing in rare disease registries and biobanks worldwide

Estrella López, Rachel Thompson, Sabina Gainotti, Chiuhui Mary Wang, Yaffa Rubinstein, Domenica Taruscio, Lucia Monaco, Hanns Lochmüller, Verónica Alonso & Manuel Posada de la Paz

Pages: 729-739

Published online: 26 May 2016

AbstractforOverview of existing initiatives to develop and improve access and data sharing in rare disease registries and biobanks worldwide | Full Text | References | PDF (964.8 KB)

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Review

Managing uveitis in patients with juvenile idiopathic arthritis

Ivan Foeldvari

Pages: 741-745

Published online: 08 Jun 2016

AbstractforManaging uveitis in patients with juvenile idiopathic arthritis | Full Text | References | PDF (533.3 KB)

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Review

Current management of Alström syndrome and recent advances in treatment

R. B. Paisey & K. Leeson-Beevers

Pages: 747-759

Published online: 02 Jun 2016

AbstractforCurrent management of Alström syndrome and recent advances in treatment | Full Text | References | PDF (1.9 MB) | Supplemental

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Review

Investigating therapies in ependymoma

Brett J. Theeler & Mark R. Gilbert

Pages: 761-773

Published online: 08 Jun 2016

AbstractforInvestigating therapies in ependymoma | Full Text | References | PDF (1.9 MB)

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Drug Evaluation

Article

Bortezomib for the treatment of acute lymphoblastic leukemia

Bruce Bostrom

Pages: 775-780

Published online: 19 May 2016

AbstractforBortezomib for the treatment of acute lymphoblastic leukemia | Full Text | References | PDF (449.7 KB)

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Article

Talimogene laherparepvec in advanced melanoma

Megan H. Pollack, Jonathan Aston, Mona Benrashid, Douglas B. Johnson & Igor Puzanov

Pages: 781-788

Published online: 19 May 2016

AbstractforTalimogene laherparepvec in advanced melanoma | Full Text | References | PDF (628.2 KB)

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Article

Evolocumab for the treatment of homozygous familial hypercholesterolaemia

Dirk Jacobus Blom & A. David Marais

Pages: 789-798

Published online: 28 May 2016

AbstractforEvolocumab for the treatment of homozygous familial hypercholesterolaemia | Full Text | References | PDF (859.9 KB)

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Erratum

Correction

Erratum

Page: 1

Published online: 17 Jun 2016

CitationforErratum | Full Text | PDF (85.9 KB)

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