ReviewDegenerative neurological and neuromuscular disease2012
Evaluation of tafamidis as first-line therapeutic agent for transthyretin familial amyloidotic polyneuropathy.
Review in Degenerative neurological and neuromuscular disease, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
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.
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.
Who cites it
3 citing papers in PubMed, 6 citations in OpenAlex.
- Development of Benziodarone Analogues with Enhanced Potency for Selective Binding to Transthyretin in Human Plasma.Journal of medicinal chemistry · 2024Article
- A Comprehensive Review on Chemistry and Biology of Tafamidis in Transthyretin Amyloidosis.Mini reviews in medicinal chemistry · 2024Review
- Mechanism of Action and Clinical Application of Tafamidis in Hereditary Transthyretin Amyloidosis.Neurology and therapy · 2016Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Almost 100 mutations in the human transthyretin (TTR) gene cause the autosomal dominant disorders of familial amyloidotic polyneuropathy (FAP) and familial amyloidotic cardiomyopathy. While these have been clinically classified as separate disorders, the peripheral and autonomic nervous systems and the heart are frequently involved in the same patient. Deposition of amyloid derived from a kinetically or thermodynamically unstable mutant TTR precursor produces an ascending sensorimotor polyneuropathy with marked autonomic involvement. Since 1990, treatment has been liver transplantation from a donor carrying two wild-type TTR genes, providing a crude form of gene therapy. Multiple studies have shown that small molecules fitting in the T
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Registered trials
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.