Disease‐Modifying Drugs Extend Survival in Hereditary Transthyretin Amyloid Polyneuropathy-Reference-Cited by-同舟云学术

Disease‐Modifying Drugs Extend Survival in Hereditary Transthyretin Amyloid Polyneuropathy

Published:2023-12-23 Issue:2 Volume:95 Page:230-236
ISSN:0364-5134
Container-title:Annals of Neurology
language:en
Short-container-title:Annals of Neurology

Author:

Ueda Mitsuharu¹²^ORCID,Misumi Yohei¹²^ORCID,Nomura Toshiya¹²,Tasaki Masayoshi¹²³,Yamakawa Shiori¹,Obayashi Konen⁴,Yamashita Taro⁵,Ando Yukio⁶

Affiliation:

1. Department of Neurology Graduate School of Medical Sciences, Kumamoto University Kumamoto Japan

2. Amyloidosis Center Kumamoto University Hospital Kumamoto Japan

3. Department of Biomedical Laboratory Sciences Graduate School of Health Sciences, Kumamoto University Kumamoto Japan

4. Department of Morphological and Physiological Sciences Graduate School of Health Sciences, Kumamoto University Kumamoto Japan

5. Department of Neurology Soyo Hospital Kumamoto Japan

6. Department of Amyloidosis Research, Faculty of Pharmaceutical Sciences Nagasaki International University Nagasaki Japan

Abstract

Hereditary transthyretin (ATTRv) amyloidosis is a rare, fatal systemic disease, associated with polyneuropathy and cardiomyopathy, that is caused by mutant transthyretin (TTR). In addition to liver transplantation, several groundbreaking disease‐modifying drugs (DMDs) such as tetrameric TTR stabilizers and TTR gene‐silencing therapies have been developed for ATTRv amyloid polyneuropathy. They were based on a working hypothesis of the mechanisms of ATTRv amyloid formation. In this retrospective cohort study, we investigated survival of all 201 consecutive patients with ATTRv amyloidosis in our center. The effects of DMDs on survival improvements were significant not only in early‐onset patients but also in late‐onset patients. ANN NEUROL 2024;95:230–236

Funder

Japan Agency for Medical Research and Development

Japan Society for the Promotion of Science

Ministry of Health, Labour and Welfare

Publisher

Wiley

Subject

Neurology (clinical),Neurology

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/ana.26845

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