Article

Real-World Effectiveness and Clinical Predictors of Response to First-Generation TTR Silencers in Variant ATTR Amyloidosis with Polyneuropathy

2026-03-18

Abstract excerpt

<title>Abstract</title> <p>Background: Transthyretin (TTR) gene-silencing therapies have transformed the management of variant transthyretin amyloidosis with polyneuropathy (ATTRv-PN). However, real-world evidence on effectiveness, safety, and durability is limited. <h4>Methods:</h4> We conducted a multicenter retrospective study across ten Spanish referral hospitals including 98 genetically confirmed ATTRv-PN pa...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: d0788bab-6e28-5d36-bf94-95dd92ded827
DOI: 10.21203/rs.3.rs-9019314/v1

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Real-World Effectiveness and Clinical Predictors of Response to First-Generation TTR Silencers in Variant ATTR Amyloidosis with PolyneuropathyDOI 10.21203/rs.3.rs-9019314/v1Read

Real-life experience with inotersen in hereditary transthyretin amyloidosis with late-onset phenotype: Data from an early-access program in Italy.Semantic proximity Read

Practical Guidance for the Use of Patisiran in the Management of Polyneuropathy in Hereditary Transthyretin-Mediated AmyloidosisSemantic proximity Read

Canadian Guidelines for Hereditary Transthyretin Amyloidosis Polyneuropathy ManagementSemantic proximity Read

Patisiran, an RNAi Therapeutic for the Treatment of Hereditary Transthyretin-Mediated AmyloidosisSemantic proximity Read

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin AmyloidosisSemantic proximity Read

Drug and Gene Therapy for Treating Variant Transthyretin Amyloidosis (ATTRv) Neuropathy.Semantic proximity Read

Treatment characteristics of patients with hereditary transthyretin amyloidosis: a cohort studySemantic proximity Read

Quality of life outcomes in APOLLO, the phase 3 trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis.Semantic proximity Read

Effect of Eplontersen in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy Across Genetic Variants: An Exploratory Analysis From the NEURO-TTRansform Trial.Semantic proximity Read

Impact of RNAi therapeutics on cardiac parameters in patients with hereditary transthyretin amyloidosis initially treated with stabilizers: A French real-world study.Semantic proximity Read

Neuropathy Assessment and Treatment Patterns in Patients with Hereditary Transthyretin AmyloidosisSemantic proximity Read

Hereditary transthyretin amyloidosis: baseline characteristics of patients in the NEURO-TTR trial.Semantic proximity Read