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ORPHA:391673

Necrotizing enterocolitis

medium confidenceDisorder

Publications

29,296

98.5th percentile

Trials

115

Interventional, condition-specific

Researchers

979

Distinct authors in sample

Gene link

—

Readiness

4/6

Stages with a signal

Clinical definition (Orphanet)

A rare intestinal disease characterized by potentially life-threatening inflammatory bowel necrosis predominantly affecting preterm neonates. Patients may present with feeding intolerance, lethargy, temperature instability, abdominal distention, blood-stained stools, diarrhea, bilious vomiting, apnea, and signs of sepsis. Radiographic features include pneumatosis intestinalis, portal venous gas, presence of fixed, dilated intestinal loops, bowel wall edema, and (in case of bowel perforation) pneumoperitoneum.

How rare: 1-5 / 10 000 — about one to five people per ten thousand (still uncommon, but less ultra-rare).

Orphanet entry

Cross-references

Joined from Mondo / Orphanet. MeSH labels may enter searches; UMLS / OMIM / NCIT are stored for reference.

Additional Mondo synonyms (2)

NEC · necrotizing enterocolitis

Research stages

Trial readiness signals

Where this condition sits on an open-data research pipeline — not how close a treatment is, and not medical advice. Empty stages often mean “not in these databases under this Mondo ID,” not “impossible.”

4/6 stages with a signal

An interventional trial matched this condition name on ClinicalTrials.gov — see trials below.

  1. Gene identifiedNot found

    No GenCC disease–gene assertion in this build

  2. LiteraturePresent

    29,296 matched papers (19,063 in last 10 years) Source

  3. Phenotype characterisedPresent

    34 HPO annotations (e.g. Decreased total neutrophil count; Abdominal distention; Poor appetite) Source

  4. Animal modelNot found

    No Alliance genotype “model of” associations via Monarch for these Mondo IDs

  5. Orphan designationPresent

    1 FDA designation (1 FDA orphan-indication approval) — e.g. Lactobacillus acidophilus and Bifidobacterium animalis subsp. lactis Source

  6. Interventional trialPresent

    115 matched on ClinicalTrials.gov (13 recruiting in sample)

Biology

Genes and phenotypes

Gene–disease validity from GenCC, plus phenotypes and animal models joined from Monarch Initiative via Mondo ID — not a clinical diagnosis aid.

Do we know what causes it?

Not yet — the cause hasn't been pinned down in GenCC.

No strong gene–disease assertion joined for this Orphanet entity.

Phenotypes (Monarch / HPO)

34

Associated phenotypes · MONDO:0005313

  • Decreased total neutrophil count
  • Abdominal distention
  • Poor appetite
  • Fatigue
  • Food intolerance

Showing 5 of 34 — open Monarch for the full list.

Animal models (Monarch / Alliance)

None returned for this Mondo ID. Empty here is not proof that no model organism work exists under another name or gene.

Monarch fetch 2026-07-29

Therapies

Designations, candidates, and chemicals

FDA OOPD and EMA orphan designations, Open Targets clinical candidates, and CTD chemical associations via MyDisease.info. These never change the interventional-trial headline.

Orphan designation (FDA · EMA)

1

Designation · 1 with FDA orphan-indication approval

  • FDA Lactobacillus acidophilus and Bifidobacterium animalis subsp. lactisNEC Very Low Birth Weight Necrotizing Enterocolitis Necrotizing enterocolitis · 2015-03-24 · Not FDA Approved for Orphan Indication

Sources: FDA OOPD · EMA orphan designations

Open Targets candidates

23

Drugs / clinical candidates · MONDO_0005313

CTD chemicals (MyDisease.info)

11 associated chemicals · 239 pathways. Therapeutic evidence is listed first when present — not a treatment recommendation.

  • Dexamethasone · therapeutic
  • Docosahexaenoic Acids · therapeutic
  • Resveratrol · therapeutic
  • Sildenafil Citrate · therapeutic
  • sodium nitrate · therapeutic
  • Clozapine · marker/mechanism
  • Gemcitabine · marker/mechanism
  • Indomethacin · marker/mechanism
  • Lipopolysaccharides · marker/mechanism
  • Oxygen · marker/mechanism
  • Vitamin E · marker/mechanism

Pathways: Glycolysis / Gluconeogenesis; Pentose and glucuronate interconversions; Fructose and mannose metabolism; Ascorbate and aldarate metabolism; Fatty acid degradation; Arginine biosynthesis; Valine, leucine and isoleucine degradation; Lysine degradation

MyDisease.info · MONDO:0005313

Literature

Is anyone studying this?

29,296

29,296 papers — among the better-studied rare conditions, though still a fraction of common-disease literature (breast cancer: over 700,000). Median papers in the last 10 years for a rare disease in this dataset (publications denominator n=3967) is 59.

29,296 papers since the earliest indexed year in this search — median last-10-year count for a rare disease in this dataset is 59 (publications denominator n=3967).

19,063 in the last 10 years · medium confidence · 98.5th percentile (publications denominator)

Phrase hits: 29,267 · MeSH hits: 137

Open Europe PMC search

Who's working on it?

979

Distinct author names in 200 sampled papers — named people below.

Who's working on it?

People publishing on this condition (sampled Europe PMC records). Affiliation is the most recent found in that sample.

  1. 01
    Garg PM9 papers · 2026

    Department of Pediatrics/Neonatology, Atrium Health Wake Forest Baptist, Wake Forest School of Medicine, Winston Salem, NC, USA.

    Papers in Europe PMC
  2. 02
    Wang J9 papers · 2026

    Department of Neonatology, Children's Hospital of Chongqing Medical University, Chongqing, China.

    Papers in Europe PMC
  3. 03
    Li Y7 papers · 2026

    Department of Neonatology, Children's Hospital of Soochow University, Suzhou, China.

    Papers in Europe PMC
  4. 04
    Liu X7 papers · 2026

    Heart Center, Guangzhou Women and Children's Medical Center, Guangzhou Medical University Guangzhou 510623, Guangdong, China.

    Papers in Europe PMC
  5. 05
    Chen Y6 papers · 2026

    Department of Pediatrics, The Second Affiliated Hospital of Guangxi Medical University, Guangxi, China.

    Papers in Europe PMC
  6. 06
    Wang Z6 papers · 2026

    Department of Neonatology, Children's Hospital of Chongqing Medical University, National Clinical Research Center for Child Health and Disorders, Ministry of Education Key Laboratory of Child Development and Disorders, Chongqing Key Laboratory of Structural Birth Defect and Reconstruction, Chongqing, China.

    Papers in Europe PMC
  7. 07
    Zhang Y6 papers · 2026

    Key Lab of Dairy Science, Ministry of Education, College of Food Science, Northeast Agricultural University, Harbin, Heilongjiang Province 150030, China.

    Papers in Europe PMC
  8. 08
    Chen J5 papers · 2026

    Neonatal Intensive Care Unit, Children's Hospital of Soochow University, Suzhou, China.

    Papers in Europe PMC
  9. 09
    Gao K5 papers · 2026

    Department of Pediatric Surgery, Affiliated Hospital of Nantong University, Nantong, Jiangsu, China.

    Papers in Europe PMC
  10. 10
    Good M5 papers · 2026

    Division of Neonatal-Perinatal Medicine, Department of Pediatrics, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA. mistygood@unc.edu.

    Papers in Europe PMC

Clinical research

Is a treatment being tested?

115

interventional trials for this specific condition

115 interventional trials matched this specific condition name; 13 currently recruiting in our sample. 36 trials are registered for enterocolitis, the broader category — shown separately because they may or may not enrol this specific subtype.

Data as of 11 September 2026

115 interventional trials — more than 77.2% of diseases in the trials denominator have none at all (5501 of 7126; this disease is at the 98.7th percentile).

medium confidence · 98.7th percentile (trials denominator)

Recruiting interventional trials

From the matched ClinicalTrials.gov set

115 interventional trials matched after quoted-phrase search and title/condition post-filter.

Broader category: enterocolitis

36

Interventional trials for the parent category, exclusive of NCT IDs already counted above. Eligibility for this subtype is not guaranteed.

Worth raising with a clinician. How we count trials.

Recruiting under the broader category

Observational and natural-history studies

86 observational studies match this condition. These do not test a treatment and are not counted in the interventional-trial headline, but they are genuine research: natural-history work often defines the endpoints needed for a future rare-disease trial, and families may be able to enroll.

Recruiting or not-yet-recruiting

Open the complete matched search on ClinicalTrials.gov

Other registries (secondary)

Broader net from EU CTIS, ISRCTN, and ICTRP when available — deduped against ClinicalTrials.gov IDs already counted above. Dual-model LLM relevance gates what we keep. These rows are not added to the interventional headline.

raw 52 · after dedupe 52 · already on CT.gov 0 · kept 0 · parent 0 · uncertain 52 · dropped 0 · fetched 2026-07-30

Source notes: ictrp: Error: ICTRP public search unavailable (WHO portal is SPA-only; SOAP needs partnership). Tried: https://apps.who.int/tri

No secondary-registry studies passed dual-model relevance for this condition name (after dedupe).

Uncertain / not reviewed (52)

Where to find support

Condition-specific patient organisations, when Orphanet lists them, are on the disease’s Orphanet page. We also link umbrella groups that support undiagnosed and ultra-rare families.

Orphanet entry for Necrotizing enterocolitis — check Associations / patient organisations on that page.

India — NPRD

Last verified 2026-07-26

This ORPHAcode is not on our curated NPRD list (direct or Mondo-parent match). That does not decide clinical eligibility; families in India should ask a notified Centre of Excellence about current coverage.

Hand-curated for this project. ORPHAcode mappings are best-effort and may be incomplete or imprecise for umbrella categories. Parent (Mondo) matches mean the policy lists a broader category — confirm eligibility with a Centre of Excellence. Financial entitlements summarised from public policy statements and may change. This is not official government guidance.

How we counted this

Europe PMC query (preferred label + any corrected label + Orphanet and Mondo exact synonyms, stoplisted; unioned with resolved MeSH labels when available). UMLS / OMIM / NCIT cross-references are stored on the overview but are not added to the query string.

"Necrotizing enterocolitis"

Run this search on Europe PMC

MeSH descriptor terms unioned into the query: Enterocolitis, Necrotizing

ClinicalTrials.gov query (quoted phrases + MeSH via query.cond, plus recall-expansion terms when used):

"Necrotizing enterocolitis" OR "Enterocolitis, Necrotizing"

Interventional trials matched via: both, phrase, mesh (mesh = registered under a MeSH descriptor no name phrase would catch; recall-expansion = gene / selected parent terms used only for trials).

Study-type breakdown: 115 interventional · 86 observational · 0 expanded access. Only interventional studies enter the trial headline.

Parent-category trials query:

"enterocolitis"

Query health: ok — strategies attempted: phrase, mesh; with hits: phrase, mesh

Run this search on ClinicalTrials.gov

Synonyms dropped by stoplist: NEC

Confidence reasoning

  • Preferred label is multi-word and distinctive
  • 1 synonym(s) dropped by stoplist (may under-count)
  • No label/synonym collisions with other diseases in this corpus

Ingested 2026-07-27T15:09:53.337Z