Evidence map›Paper›PMID 42814300›Full record

ReviewMethods in molecular biology (Clifton, N.J.)2026

Inotersen for the Treatment of Hereditary Transthyretin Amyloidosis.

Maria Mahfouz, Alishba Raza, Rika Maruyama, Toshifumi Yokota

Abstract readReview
PubMed Publisher
In one paragraph

Review in Methods in molecular biology (Clifton, N.J.), 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

4 authors.

Maria MahfouzDepartment of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB, Canada, T6G 2H7.
Alishba RazaDepartment of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB, Canada, T6G 2H7.
Rika MaruyamaDepartment of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB, Canada, T6G 2H7.
Toshifumi YokotaDepartment of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB, Canada, T6G 2H7. toshifum@ualberta.ca.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Hereditary transthyretin amyloidosis (hATTR) is a rare autosomal dominant condition in which mutations in the transthyretin gene cause amyloid fibrils to develop and deposit into tissues, affecting primarily the nerves and heart, causing polyneuropathy and cardiomyopathy, respectively. Standard treatment has been liver transplants to try to eliminate the mutated transthyretin products, as the liver is the main source of transthyretin production. Inotersen (brand name Tegsedi), also known as IONIS-TTR

Indexed as

Amyloid Neuropathies, FamilialOligonucleotidesPrealbuminAnimalsHumansMutationOligonucleotides, AntisenseInotersenOligonucleotidesOligonucleotides, AntisensePrealbumin2’-O-methoxyethyl (2’-MOE)Antisense oligonucleotides (ASOs)European CommissionFamilial amyloidosis neuropathy (FAP)Food and Drug Administration (FDA)Health CanadaHereditary transthyretin amyloidosis (hATTR)Inotersen (Tegsedi)PatisiranRibonuclease H1 (RNase H1)

Identifiers

PMID42814300

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.