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ORPHA:439175

Pediatric arterial ischemic stroke

high confidenceDisorder

Also known as: Childhood AIS · Childhood arterial ischemic stroke · Pediatric AIS

Publications

660

81.7th percentile

Trials

1

Interventional, condition-specific

Researchers

1,058

Distinct authors in sample

Gene link

—

Readiness

3/6

Stages with a signal

Clinical definition (Orphanet)

A rare neurologic condition characterized by focal cerebral ischemia and infarction due to blockage of a brain artery with subsequent impairment of blood supply and oxygenation of brain tissue. Most children present with hemiparesis with or without facial palsy at stroke onset. In addition, compared to adults, children more often suffer strokes in the posterior circulation, leading to or oculomotor disturbance. Likewise, aphasia is more frequent in pediatric patients. Other signs and symptoms include , headache, vomiting, and alterations in the level of consciousness. Children under one year of age are more likely to present with and altered level of consciousness, while older children more often show focal neurological deficits.

Orphanet entry

Cross-references

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

Additional Mondo synonyms (5)

childhood AIS · childhood arterial ischaemic stroke · childhood arterial ischemic stroke · paediatric AIS · pediatric AIS

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.”

3/6 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

    660 matched papers (432 in last 10 years) Source

  3. Phenotype characterisedPresent

    1 HPO annotations (e.g. Childhood onset) Source

  4. Animal modelNot found

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

  5. Orphan designationNot found

    No FDA or EMA orphan-drug designation matched this disease via UMLS or preferred name Source

  6. Interventional trialPresent

    1 matched on ClinicalTrials.gov

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)

1

Associated phenotypes · MONDO:0018585

  • Childhood onset

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)

No designation matched this disease via UMLS or preferred name on the FDA OOPD mirror or EMA orphan register. Absence here is not proof that none exists under another wording.

Open Targets candidates

No drugs or clinical candidates returned for this Mondo ID on Open Targets.

CTD chemicals (MyDisease.info)

No CTD chemical associations returned for this Mondo ID.

Literature

Is anyone studying this?

660

660 papers have been published on this condition. That is a real research literature — still far smaller than common diseases (breast cancer: over 700,000 papers). Median papers in the last 10 years for a rare disease in this dataset (publications denominator n=3967) is 59.

660 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).

432 in the last 10 years · high confidence · 81.7th percentile (publications denominator)

Phrase hits: 660 · MeSH hits: 0

Open Europe PMC search

Who's working on it?

1,058

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
    Dlamini N16 papers · 2026

    From Department of Neurology Columbia University, New York, NY (M.S.V.E.); Department of Epidemiology, Mailman School of Public Health, New York, NY (M.S.V.E.); Departments of Neurology (N.K.H., H.J.F.), Biostatistics & Epidemiology (N.K.H.), and Pediatrics (C.A.G., H.J.F.), University of California San Francisco, San Francisco, CA; Department of Pediatrics (Infectious Disease), Kaiser Permanente, Oakland, CA (C.A.G.); Departments of Pediatrics and Neurology, Ohio State University, Columbus OH (W.D.L.); Department of Neurology, University of Washington, Seattle, WA (C.A.-L,); Department of Neurology, Hospital for Sick Children, Toronto, Canada (N.D., G.A.deV.); Department of Neurology, Alder Hey Children's NHS Foundation Trust, Liverpool, England (R.K.); Department of Pathology, Columbia University, New York, NY (E.A.H.); and Department of Radiology, Stanford University, Palo Alto, CA (M.W.).

    Papers in Europe PMC
  2. 02
    Fullerton HJ16 papers · 2026

    From Department of Neurology Columbia University, New York, NY (M.S.V.E.); Department of Epidemiology, Mailman School of Public Health, New York, NY (M.S.V.E.); Departments of Neurology (N.K.H., H.J.F.), Biostatistics & Epidemiology (N.K.H.), and Pediatrics (C.A.G., H.J.F.), University of California San Francisco, San Francisco, CA; Department of Pediatrics (Infectious Disease), Kaiser Permanente, Oakland, CA (C.A.G.); Departments of Pediatrics and Neurology, Ohio State University, Columbus OH (W.D.L.); Department of Neurology, University of Washington, Seattle, WA (C.A.-L,); Department of Neurology, Hospital for Sick Children, Toronto, Canada (N.D., G.A.deV.); Department of Neurology, Alder Hey Children's NHS Foundation Trust, Liverpool, England (R.K.); Department of Pathology, Columbia University, New York, NY (E.A.H.); and Department of Radiology, Stanford University, Palo Alto, CA (M.W.). Heather.Fullerton@ucsf.edu.

    Papers in Europe PMC
  3. 03
    Steinlin M16 papers · 2026

    Division of Neuropaediatrics, Development and Rehabilitation, Department of Paediatrics (R.M., I.S.-A., M.R., G.O., S.G., M.S.), Inselspital, Bern University Hospital, University of Bern, Switzerland.

    Papers in Europe PMC
  4. 04
    Lee S15 papers · 2026

    Division of Child Neurology, Department of Neurology, Stanford University, Palo Alto, CA, USA.

    Papers in Europe PMC
  5. 05
    Hills NK13 papers · 2026

    From Department of Neurology Columbia University, New York, NY (M.S.V.E.); Department of Epidemiology, Mailman School of Public Health, New York, NY (M.S.V.E.); Departments of Neurology (N.K.H., H.J.F.), Biostatistics & Epidemiology (N.K.H.), and Pediatrics (C.A.G., H.J.F.), University of California San Francisco, San Francisco, CA; Department of Pediatrics (Infectious Disease), Kaiser Permanente, Oakland, CA (C.A.G.); Departments of Pediatrics and Neurology, Ohio State University, Columbus OH (W.D.L.); Department of Neurology, University of Washington, Seattle, WA (C.A.-L,); Department of Neurology, Hospital for Sick Children, Toronto, Canada (N.D., G.A.deV.); Department of Neurology, Alder Hey Children's NHS Foundation Trust, Liverpool, England (R.K.); Department of Pathology, Columbia University, New York, NY (E.A.H.); and Department of Radiology, Stanford University, Palo Alto, CA (M.W.).

    Papers in Europe PMC
  6. 06
    Mackay MT12 papers · 2025

    Emergency Research Group and Neuroscience Research Groups Murdoch Childrens Research Institute, Melbourne, Victoria, Australia.

    Papers in Europe PMC
  7. 07
    Everts R11 papers · 2026

    Division of Neuropaediatrics, Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital, University of Bern, Switzerland.

    Papers in Europe PMC
  8. 08
    Wintermark M11 papers · 2026

    From Department of Neurology Columbia University, New York, NY (M.S.V.E.); Department of Epidemiology, Mailman School of Public Health, New York, NY (M.S.V.E.); Departments of Neurology (N.K.H., H.J.F.), Biostatistics & Epidemiology (N.K.H.), and Pediatrics (C.A.G., H.J.F.), University of California San Francisco, San Francisco, CA; Department of Pediatrics (Infectious Disease), Kaiser Permanente, Oakland, CA (C.A.G.); Departments of Pediatrics and Neurology, Ohio State University, Columbus OH (W.D.L.); Department of Neurology, University of Washington, Seattle, WA (C.A.-L,); Department of Neurology, Hospital for Sick Children, Toronto, Canada (N.D., G.A.deV.); Department of Neurology, Alder Hey Children's NHS Foundation Trust, Liverpool, England (R.K.); Department of Pathology, Columbia University, New York, NY (E.A.H.); and Department of Radiology, Stanford University, Palo Alto, CA (M.W.).

    Papers in Europe PMC
  9. 09
    Sun LR10 papers · 2026

    Department of Neurology, Johns Hopkins School of Medicine, Johns Hopkins University, Baltimore, Maryland.

    Papers in Europe PMC
  10. 10
    Amlie-Lefond C9 papers · 2026

    From Department of Neurology Columbia University, New York, NY (M.S.V.E.); Department of Epidemiology, Mailman School of Public Health, New York, NY (M.S.V.E.); Departments of Neurology (N.K.H., H.J.F.), Biostatistics & Epidemiology (N.K.H.), and Pediatrics (C.A.G., H.J.F.), University of California San Francisco, San Francisco, CA; Department of Pediatrics (Infectious Disease), Kaiser Permanente, Oakland, CA (C.A.G.); Departments of Pediatrics and Neurology, Ohio State University, Columbus OH (W.D.L.); Department of Neurology, University of Washington, Seattle, WA (C.A.-L,); Department of Neurology, Hospital for Sick Children, Toronto, Canada (N.D., G.A.deV.); Department of Neurology, Alder Hey Children's NHS Foundation Trust, Liverpool, England (R.K.); Department of Pathology, Columbia University, New York, NY (E.A.H.); and Department of Radiology, Stanford University, Palo Alto, CA (M.W.).

    Papers in Europe PMC

Clinical research

Is a treatment being tested?

1

interventional trials for this specific condition

1 interventional trial matched this specific condition name; none in our sample are currently recruiting. 1,561 trials are registered for ischemic stroke, the broader category — shown separately because they may or may not enrol this specific subtype.

Data as of 11 September 2026

1 interventional trial — more than 77.2% of diseases in the trials denominator have none at all (5501 of 7126; this disease is at the 80.1th percentile).

high confidence · 80.1th percentile (trials denominator)

Recruiting interventional trials

From the matched ClinicalTrials.gov set

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

No currently recruiting studies in the matched set. Open the same search on ClinicalTrials.gov.

Broader category: ischemic stroke

1,561

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

1 observational study matches 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.

None of the matched observational studies is currently listed as 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 5 · after dedupe 5 · already on CT.gov 0 · kept 0 · parent 0 · uncertain 5 · 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 (5)

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 Pediatric arterial ischemic stroke — 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.

"Pediatric arterial ischemic stroke" OR "Childhood AIS" OR "Childhood arterial ischemic stroke" OR "Pediatric AIS" OR "childhood arterial ischaemic stroke" OR "paediatric AIS"

Run this search on Europe PMC

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

"Pediatric arterial ischemic stroke" OR "Childhood AIS" OR "Childhood arterial ischemic stroke" OR "Pediatric AIS" OR "childhood arterial ischaemic stroke" OR "paediatric AIS"

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

Study-type breakdown: 1 interventional · 1 observational · 0 expanded access. Only interventional studies enter the trial headline.

Parent-category trials query:

"ischemic stroke"

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

Run this search on ClinicalTrials.gov

Confidence reasoning

  • Preferred label is multi-word and distinctive
  • No synonyms dropped by stoplist
  • No label/synonym collisions with other diseases in this corpus

Ingested 2026-07-27T16:16:53.076Z