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Volume 11, Issue 1

Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 11, Issue 1 (2023)

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Expert Opinion on Orphan Drugs, Volume 11, Issue 1 (2023)

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Vol 12, 2024 Volume 11, 2023 Vol 10, 2022 Vol 9, 2021 Vol 8, 2020 Vol 7, 2019 Vol 6, 2018 Vol 5, 2017 Vol 4, 2016 Vol 3, 2015 Vol 2, 2014 Vol 1, 2013

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Original Research

Article

Recommendations for the management of diarrhea with trofinetide use in Rett syndrome

Eric D. Marsh, Arthur Beisang, Timothy Buie, Timothy A. Benke, Brian Gaucher & Kathleen J. Motil

Pages: 1-8

Published online: 13 Jun 2023

AbstractforRecommendations for the management of diarrhea with trofinetide use in Rett syndrome | Full Text | References | PDF (285.3 KB) | EPUB

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Open Access

Perspective

Real world data for rare diseases research: The beginner’s guide to registries

Federica Pisa, Ariel Arias, Emily Bratton, Maribel Salas & Janet Sultana

Pages: 9-15

Published online: 01 Aug 2023

AbstractforReal world data for rare diseases research: The beginner’s guide to registries | Full Text | References | PDF (276.8 KB) | EPUB

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Open Access

Review

Review Article

Overview of genetic testing in Prader-Willi syndrome

Ying Gao, Mian-Lin Zhong, Yang-Li Dai, Yong-Hui Jiang & Chao-Chun Zou

Pages: 16-25

Published online: 28 Sep 2023

AbstractforOverview of genetic testing in Prader-Willi syndrome | Full Text | References | PDF (1.2 MB) | EPUB

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Orphan drugs approved in Canada: health technology assessment, price negotiation, and government formulary listing

Nigel S B. Rawson et al.

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Overview of genetic testing in Prader-Willi syndrome

Ying Gao et al.

Review Article | Published online: 28 Sep 2023

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Expert Opinion on Orphan Drugs (Vol.11, Iss.1, 2023)

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