ReviewCureus2026
Real-World Experience With Givosiran in Acute Porphyrias: A Narrative Review and a Novel Hypothesis.
Review in Cureus, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
What it found
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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
2 citing papers in PubMed.
- A Case Report of Acute Intermittent Porphyria Accompanied by Severe Peripheral Neuropathy.Diagnostics (Basel, Switzerland) · 2026Article
- Target, silence, replace: a review on RNA-based drugs in modern medicine.Frontiers in cell and developmental biology · 2026Review
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.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Real-world experience with givosiran has been accumulating after its approval for the treatment of patients with acute porphyria and chronic attacks in the United States and in Europe, respectively. Up to now, nearly as many patients from various countries have been treated after the registration of the drug and reported in real-world studies as treated in the randomized phase 3 ENVISION trial. Most reports confirm high drug efficiency, but some also report adverse effects (homocysteinemia, lipase elevation, and kidney function impairment). Moreover, many patients suffer from breakthrough attacks, which remain a conundrum. Since gallbladder epithelial cells also contain the asialoglycoprotein receptor, which is a prerequisite for the uptake of givosiran, it could be that the siRNA-induced depletion of haem leads to a disturbance of the function of the gallbladder. Therefore, we hypothesize a potential role of the gallbladder and the metabolism of bile acids in the development of porphyria and givosiran-resistant attacks, which has to be supported by clinical or experimental validation.
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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.