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Future Rare Diseases

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Future Rare Diseases, Volume 3, Issue 4 (2023)

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Future Rare Diseases, Volume 3, Issue 4 (2023)

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Vol 4, 2024 Volume 3, 2023 Vol 2, 2022 Vol 1, 2021

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Plain Language Summary of Publication

Treatment with Marstacimab for People with Severe Hemophilia a or B: a Plain Language Summary of the results from a short-term study and a longer-term study

Johnny N Mahlangu, Jose Luis Lamas, Juan Cristobal Morales, Daniel R Malan, Silva Zupančić Šalek, Michael Wang, Lisa N Boggio, Inga Hegemann, Andrzej Mital, Matthew Cardinal, Tong Zhu, Pengling Sun, John Teeter, Robert J Charnigo, Eunhee Hwang & Steven Arkin

Article: FRD45

Published online: 07 Sep 2023

First Page PreviewforTreatment with Marstacimab for People with Severe Hemophilia a or B: a Plain Language Summary of the results from a short-term study and a longer-term study | PDF (334.9 KB)

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Plain Language Summary of Publication

A Plain Language Summary of Results from the LAVENDER Study: Trofinetide Treatment for Rett Syndrome

Jeffrey L Neul, Alan K Percy, Timothy A Benke, Elizabeth M Berry-Kravis, Daniel G Glaze, Victor Abler, Tim Lin, Kathie M Bishop & James M Youakim

Article: FRD44

Published online: 08 Aug 2023

First Page PreviewforA Plain Language Summary of Results from the LAVENDER Study: Trofinetide Treatment for Rett Syndrome | PDF (695.6 KB)

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Commentary

Editorial

Genomics in Rare Diseases: An Overview for the Patient, Family and Non-Specialist Healthcare Professional

Alastair Kent, Alasdair PJ Parker, Arti Patel, Sarah L Wynn & Charles A Steward

Article: FRD56

Published online: 13 Dec 2023

First Page PreviewforGenomics in Rare Diseases: An Overview for the Patient, Family and Non-Specialist Healthcare Professional | Full Text | References | PDF (223.8 KB) | EPUB

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Interview

Discussion

Creld1 Warriors: Arming Parents with the Support They Need

Adam Clatworthy

Article: FRD55

Published online: 07 Dec 2023

AbstractforCreld1 Warriors: Arming Parents with the Support They Need | Full Text | PDF (143.2 KB) | EPUB

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Patient Perspective

Living With Lung Fibrosis: Knowledge is Power

Bill Vick, Patti Vick & Haley Hoy

Article: FRD51

Published online: 17 Nov 2023

AbstractforLiving With Lung Fibrosis: Knowledge is Power | Full Text | References | PDF (1.1 MB) | EPUB

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Plain Language Summary of Publication

Plain language summary of the GENEr8-1 clinical trial of valoctocogene roxaparvovec gene therapy for hemophilia A

Gillian Lowe, Simon Fletcher, Patrick James Lynch, Johnny Mahlangu, Margareth C Ozelo, Luke Pembroke, Steven W Pipe, Gabriela G Yamaguti-Hayakawa, Deon York, Tara M Robinson, Hua Yu & Leonard A Valentino

Article: FRD46

Published online: 02 Nov 2023

First Page PreviewforPlain language summary of the GENEr8-1 clinical trial of valoctocogene roxaparvovec gene therapy for hemophilia A | PDF (799.6 KB)

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Future Rare Diseases (Vol.3, Iss.4, 2023)

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