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Volume 6, Issue 3

Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 6, Issue 3 (2018)

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Expert Opinion on Orphan Drugs, Volume 6, Issue 3 (2018)

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Editorial

Lessons from chylomicron retention disease: a potential new approach for the treatment of hypercholesterolemia?

Noel Peretti

Pages: 163-165

Published online: 12 Feb 2018

First Page PreviewforLessons from chylomicron retention disease: a potential new approach for the treatment of hypercholesterolemia? | Full Text | References | PDF (312 KB)

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Review

Gene therapy for the treatment of X-linked retinitis pigmentosa

Cristina Martinez-Fernandez De La Camara, Anika Nanda, Anna Paola Salvetti, M. Dominik Fischer & Robert E. MacLaren

Pages: 167-177

Published online: 27 Feb 2018

AbstractforGene therapy for the treatment of X-linked retinitis pigmentosa | Full Text | References | PDF (1.1 MB)

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Review

The potential of utrophin modulators for the treatment of Duchenne muscular dystrophy

Simon Guiraud, David Roblin & Davies. E. Kay

Pages: 179-192

Published online: 15 Feb 2018

AbstractforThe potential of utrophin modulators for the treatment of Duchenne muscular dystrophy | Full Text | References | PDF (1.5 MB)

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Review

Gene therapy strategies for X-linked myotubular myopathy

Christopher R. Pierson

Pages: 193-202

Published online: 26 Feb 2018

AbstractforGene therapy strategies for X-linked myotubular myopathy | Full Text | References | PDF (785.9 KB)

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Review

Advances in the understanding of hereditary ataxia – implications for future patients

Anna Zeitlberger, Heather Ging, Suran Nethisinghe & Paola Giunti

Pages: 203-217

Published online: 14 Mar 2018

AbstractforAdvances in the understanding of hereditary ataxia – implications for future patients | Full Text | References | PDF (1.1 MB)

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Review

Clinical trial design for Friedreich ataxia - Where are we now and what do we need?

Christian Rummey, Elizabeth Kichula & David R. Lynch

Pages: 219-230

Published online: 14 Mar 2018

AbstractforClinical trial design for Friedreich ataxia - Where are we now and what do we need? | Full Text | References | PDF (1.1 MB)

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Drug Evaluation

Article

Developing role of eltrombopag in the treatment of aplastic anemia

Phillip Scheinberg

Pages: 231-235

Published online: 26 Feb 2018

AbstractforDeveloping role of eltrombopag in the treatment of aplastic anemia | Full Text | References | PDF (457.5 KB)

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Article

Human plasma-derived C1 esterase inhibitor for on-demand or prophylaxis treatment of patients with hereditary angioedema: intravenous and subcutaneous formulations

Jonathan A. Bernstein

Pages: 237-245

Published online: 23 Feb 2018

AbstractforHuman plasma-derived C1 esterase inhibitor for on-demand or prophylaxis treatment of patients with hereditary angioedema: intravenous and subcutaneous formulations | Full Text | References | PDF (921.3 KB)

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