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Parathyroid Disorders and Treatments / Alzheimer's Disease Research and Treatments · Journal article
Neurology and Therapy · July 15, 2026
A consensus or society position rather than new primary data.
This is a narrative clinical review of disease-modifying therapies for hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), covering TTR stabilizers, RNA-silencing agents, and emerging gene-editing approaches. It synthesizes evidence from clinical trials to guide therapeutic selection but does not report original data or quantitative comparisons.
Journal article. Patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), an autosomal dominant disorder caused by pathogenic variants in the TTR gene.
TTR stabilizers include tafamidis, diflunisal, and acoramidis RNA-silencing agents (patisiran, vutrisiran, inotersen, eplontersen) reduce hepatic TTR production and slow disease progression Clinical trials of RNA interference therapies demonstrated significant improvements in neuropathy impairment scores, quality of life, and functional outcomes with acceptable safety profiles
Long-term safety and durability data for newer agents not specified with numbers Clinical trials of RNA interference therapies demonstrated significant improvements in neuropathy impairment scores, quality of life, and functional outcomes with acceptable safety profiles
This review provides clinicians with a comprehensive overview of available disease-modifying therapies for ATTRv-PN and their mechanisms, though it does not report quantitative comparative efficacy data to guide treatment selection in individual patients. The identification of unmet needs—including treatment costs, accessibility, and sequencing strategies—highlights areas where further real-world evidence is needed.
This is a narrative review summarizing current disease-modifying therapies for ATTRv-PN, comparing mechanisms and clinical evidence to guide clinical practice, rather than reporting original research findings.
This review provides clinicians with a comprehensive overview of available disease-modifying therapies for ATTRv-PN and their mechanisms, though it does not report quantitative comparative efficacy data to guide treatment selection in individual patients. The identification of unmet needs—including treatment costs, accessibility, and sequencing strategies—highlights areas where further real-world evidence is needed.
Graded across the dimensions that decide whether you should act, each from what the source actually supports. There is no single score, and where a dimension was not assessed it says so.
What is missing. This record has no reported figures. That is a gap in the analysis, not a judgement about the study.
Hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) is an autosomal dominant disorder caused by pathogenic variants in the transthyretin (TTR) gene resulting in progressive sensorimotor neuropathy, autonomic dysfunction, and effects on other organs, particularly the heart. Historically, treatment options have been limited to liver transplantation and symptomatic management, and the disease was associated with poor prognosis and progressive disability. However, over the past decade, the therapeutic landscape has been transformed by the development of disease-modifying therapies that target the underlying pathophysiology of TTR amyloid formation. These therapies include TTR stabilizers, such as tafamidis, diflunisal, and acoramidis, as well as RNA-silencing agents, such as patisiran, vutrisiran, inotersen, and eplontersen, which reduce hepatic production of TTR and slow disease progression. Clinical trials of RNA interference therapies have demonstrated significant improvements in neuropathy impairment scores, quality of life, and functional outcomes, with acceptable safety profiles and sustained long-term benefits. Emerging gene-editing approaches, including CRISPR-based therapies like NTLA-2001, may provide a one-time treatment and long-term disease control, representing a promising future direction for managing ATTRv-PN. Despite these advances, important challenges remain. These include high treatment costs, limited global accessibility, uncertainty regarding optimal treatment selection and sequencing, and the need for long-term real-world, patient-centered outcome data. This review summarizes current disease-modifying therapies for ATTRv-PN, compares their mechanisms and clinical evidence, and discusses unmet needs and future research directions, with the aim of providing a practical and comprehensive overview for clinicians treating patients with ATTRv-PN.
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