Evidence map›Paper›PMID 38772406›Full record

ReviewSeminars in liver disease2024

Understanding Hepatic Porphyrias: Symptoms, Treatments, and Unmet Needs.

Oluwashanu Balogun, Kari Nejak-Bowen

Abstract readReview
In one paragraph

Review in Seminars in liver disease, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Article
  2. Review
  3. Review
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

2 authors.

Oluwashanu BalogunDepartment of Experimental Pathology, University of Pittsburgh, Pittsburgh, Pennsylvania.
Kari Nejak-BowenDepartment of Experimental Pathology, University of Pittsburgh, Pittsburgh, Pennsylvania.

Funding

Beta-catenin inhibition as a novel therapeutic strategy for porphyriaR01DK124412 · NIDDK · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · PI NEJAK-BOWEN, KARI N · 2020 to 2024
$2.2M
NIDDK NIH HHS R01 DK124412NIH HHS 1R01DK124412
6 · The paper itself

Abstract

Hepatic porphyrias are a group of metabolic disorders that are characterized by overproduction and accumulation of porphyrin precursors in the liver. These porphyrins cause neurologic symptoms as well as cutaneous photosensitivity, and in some cases patients can experience life-threatening acute neurovisceral attacks. This review describes the acute hepatic porphyrias in detail, including acute intermittent porphyria, hereditary coproporphyria, and variegate porphyria, as well as the hepatic porphyrias with cutaneous manifestations such as porphyria cutanea tarda and hepatoerythropoietic porphyria. Each section will cover disease prevalence, clinical manifestations, and current therapies, including strategies to manage symptoms. Finally, we review new and emerging treatment modalities, including gene therapy through use of adeno-associated vectors and chaperone therapies such as lipid nanoparticle and small interfering RNA-based therapeutics.

Indexed as

Genetic TherapyPorphyrias, HepaticAnimalsHumans

Identifiers

PMID38772406
PMCPMC11268267

What Socratic holds

Textmetadata
LicenceTDM
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 Socratic graph.