Evidence mapPaperPMID 26747865Full record

ReviewDisease models & mechanisms2016

The role of enterocyte defects in the pathogenesis of congenital diarrheal disorders.

Arend W Overeem, Carsten Posovszky, Edmond H M M Rings, Ben N G Giepmans, Sven C D van IJzendoorn

Registry-linked trialOpen access · goldAbstract readReview
In one paragraph

Review in Disease models & mechanisms, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06721871 (Evaluation of Safety, Tolerability and Efficacy of Crofelemer Following Multiple Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Participants With Microvillus Inclusion Disease), which is not on this map. Cited by 20 papers.

0numbers the graph read from it
0cells of the map it votes in
20citing papers in PubMed
6.9field-weighted citation impact, top 3% of its field
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.

NCT06721871 phase2 / phase3active not recruitingnot on this mapstarted 2025, after this paper: background citation

Evaluation of Safety, Tolerability and Efficacy of Crofelemer Following Multiple Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Participants With Microvillus Inclusion Disease (MVID)

TypeinterventionalSponsorNapo Pharmaceuticals, Inc.Ran2025 to 2026Enrolled6ConditionsMicrovillus Inclusion Disease, Congenital Disorders, Rare DiseasesArmsCrofelemer Powder for Oral Solution, Placebo Powder for Oral Solution
3 · Its place in the literature

Who cites it

20 citing papers in PubMed, 42 citations in OpenAlex.

  1. Review
  2. Article
  3. Article
  4. Article
  5. Article
  6. Review
  7. Article
  8. Review
  9. Article
  10. Review
  11. Review
  12. Microtubule Motors in Establishment of Epithelial Cell Polarity.Cold Spring Harbor perspectives in biology · 2018
    Review
  13. Dynamic Formation of Microvillus Inclusions During Enterocyte Differentiation inCellular and molecular gastroenterology and hepatology · 2018
    Article
  14. The Endosomal Protein Endotubin Is Required for Enterocyte Differentiation.Cellular and molecular gastroenterology and hepatology · 2018
    Article
  15. Article
  16. Article
  17. Review
  18. Article
  19. Review
  20. 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

5 authors at 4 institutions in 2 countries.

Arend W OvereemDepartment of Cell Biology, University Medical Center Groningen, University of Groningen, 9713 AV Groningen, The Netherlands.
Carsten PosovszkyDepartment of Pediatrics and Adolescent Medicine, University Medical Center Ulm, 89075 Ulm, Germany.
Edmond H M M RingsDepartment of Pediatrics, Erasmus Medical Center Rotterdam, Erasmus University Rotterdam, 3000 CB Rotterdam, The Netherlands Department of Pediatrics, Leiden University Medical Center, Leiden University, 2300 RC Leiden, The Netherlands.
Ben N G GiepmansDepartment of Cell Biology, University Medical Center Groningen, University of Groningen, 9713 AV Groningen, The Netherlands.
Sven C D van IJzendoornDepartment of Cell Biology, University Medical Center Groningen, University of Groningen, 9713 AV Groningen, The Netherlands s.c.d.van.ijzendoorn@umcg.nl.
University of Groningen · NLLeiden University Medical Center · NLUniversity Hospital Ulm · DEUniversity Medical Center Groningen · NL

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Congenital diarrheal disorders are rare, often fatal, diseases that are difficult to diagnose (often requiring biopsies) and that manifest in the first few weeks of life as chronic diarrhea and the malabsorption of nutrients. The etiology of congenital diarrheal disorders is diverse, but several are associated with defects in the predominant intestinal epithelial cell type, enterocytes. These particular congenital diarrheal disorders (CDD(ENT)) include microvillus inclusion disease and congenital tufting enteropathy, and can feature in other diseases, such as hemophagocytic lymphohistiocytosis type 5 and trichohepatoenteric syndrome. Treatment options for most of these disorders are limited and an improved understanding of their molecular bases could help to drive the development of better therapies. Recently, mutations in genes that are involved in normal intestinal epithelial physiology have been associated with different CDD(ENT). Here, we review recent progress in understanding the cellular mechanisms of CDD(ENT). We highlight the potential of animal models and patient-specific stem-cell-based organoid cultures, as well as patient registries, to integrate basic and clinical research, with the aim of clarifying the pathogenesis of CDD(ENT) and expediting the discovery of novel therapeutic strategies.

Indexed as

AbetalipoproteinemiaAnimalsChylomicronsDiarrheaDiarrhea, InfantileEnterocytesFaciesFetal Growth RetardationHair DiseasesHeterozygoteHumansHypobetalipoproteinemiasLipidsMiceMice, KnockoutMicrovilliChylomicronsLipidsBrush borderCell polarityCongenital diarrheal disorderEnterocyteIntracellular traffickingMicrovillus inclusion diseases

Identifiers

PMID26747865
PMCPMC4728335
OpenAlexW2277484184

What Socratic holds

Textmetadata
LicenceCC BY
Read underepoch 390

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 Socratic graph.