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Volume 3, Issue 3

Future Rare Diseases

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Future Rare Diseases, Volume 3, Issue 3 (2023)

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Future Rare Diseases, Volume 3, Issue 3 (2023)

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Vol 4, 2023 Volume 3, 2023 Vol 2, 2022 Vol 1, 2021

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Summary

Plain Language Summary of Publication

Plain Language Summary of PEGASUS, a Study Comparing Pegcetacoplan with Eculizumab for 16 Weeks in People with Paroxysmal Nocturnal Hemoglobinuria

Morag Griffin, Jeff Szer, Ilene Weitz, Alexander Röth, Britta Höchsmann, Jens Panse, Kensuke Usuki, Peter Hillmen, Jean-Jacques Kiladjian, Carlos de Castro, Hisakazu Nishimori, Lisa Tan, Pascal Deschatelets, Cedric Francois, Federico Grossi, Temitayo Ajayi, Antonio Risitano & Régis Peffault de la Tour

Article: FRD42

Published online: 23 Jul 2023

First Page PreviewforPlain Language Summary of PEGASUS, a Study Comparing Pegcetacoplan with Eculizumab for 16 Weeks in People with Paroxysmal Nocturnal Hemoglobinuria | PDF (1.5 MB)

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Plain Language Summary of Publication

Recommendations for Managing Diarrhea from Trofinetide use in Individuals with Rett Syndrome: A Plain Language Summary

Kathleen J Motil, Arthur Beisang, Timothy A Benke, Brian Gaucher, Victor Abler & Dominique Pichard

Article: FRD43

Published online: 25 Jul 2023

AbstractforRecommendations for Managing Diarrhea from Trofinetide use in Individuals with Rett Syndrome: A Plain Language Summary | PDF (1.5 MB)

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Short Communication

Discussion

Characteristics, Conditions, and Healthcare Service Utilization Patterns of Individuals Diagnosed with Rett Syndrome: An Analysis of Administrative Claims Data

Tigwa Davis, Purva Parab, Damian M May & Charles Ruetsch

Article: FRD41

Published online: 12 Jul 2023

AbstractforCharacteristics, Conditions, and Healthcare Service Utilization Patterns of Individuals Diagnosed with Rett Syndrome: An Analysis of Administrative Claims Data | Full Text | References | PDF (675.2 KB) | EPUB

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Brief Report

Understanding Characteristics, Conditions and Treatment Patterns among Males Diagnosed with Rett Syndrome: An Analysis of Commercial Health Insurance Claims

Tigwa Davis, Purva Parab, Damian M May & Charles Ruetsch

Article: FRD40

Published online: 12 Jun 2023

AbstractforUnderstanding Characteristics, Conditions and Treatment Patterns among Males Diagnosed with Rett Syndrome: An Analysis of Commercial Health Insurance Claims | Full Text | References | PDF (1 MB) | EPUB

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Future Rare Diseases (Vol.3, Iss.3, 2023)

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