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ORPHA:2382

Lennox-Gastaut syndrome

medium confidenceDisorder

Publications

7,884

94.9th percentile

Trials

49

Interventional, condition-specific

Researchers

1,152

Distinct authors in sample

Gene link

MAPK10

Limited

Readiness

6/6

Stages with a signal

Clinical definition (Orphanet)

A rare, severe early-onset developmental epileptic characterized by the triad of intellectual impairment, multiple seizure types, and typical electroencephalography (EEG) abnormalities.

How rare: 1-9 / 1 000 000 — roughly one to nine people per million. In a city the size of Kolkata, perhaps a few dozen.

Orphanet entry

Cross-references

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

Additional Mondo synonyms (1)

LGS

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.”

6/6 stages with a signal

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

  1. Gene identifiedPresent

    Limited — MAPK10

  2. LiteraturePresent

    7,884 matched papers (5,419 in last 10 years) Source

  3. Phenotype characterisedPresent

    78 HPO annotations (e.g. Aggressive behavior; Bilateral tonic-clonic seizure; EEG abnormality) Source

  4. Animal modelPresent

    3 genotype models (Mus musculus) Source

  5. Orphan designationPresent

    11 FDA · 4 EMA designations (5 FDA orphan-indication approvals) — e.g. (-)-huperzine A Source

  6. Interventional trialPresent

    49 matched on ClinicalTrials.gov (9 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?

Possibly — only limited evidence so far for MAPK10.

GenCC classification: Limited.

Phenotypes (Monarch / HPO)

78

Associated phenotypes · MONDO:0016532

  • Aggressive behavior
  • Bilateral tonic-clonic seizure
  • EEG abnormality
  • Falls
  • Atonic seizure

Showing 5 of 78 — open Monarch for the full list.

Animal models (Monarch / Alliance)

3

Model associations linked to this Mondo ID

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)

15

Designations · 5 with FDA orphan-indication approval

  • FDA (-)-huperzine ALennox-Gastaut syndrome · 2020-06-15 · Not FDA Approved for Orphan Indication
  • FDA carisbamateLennox-Gastaut syndrome · 2017-08-02 · Not FDA Approved for Orphan Indication
  • FDA fenfluramineLennox-Gastaut syndrome · 2017-06-19 · Not FDA Approved for Orphan Indication
  • FDA cannabidiolLennox-Gastaut syndrome · 2014-06-23 · Not FDA Approved for Orphan Indication
  • FDA perampanelLennox-Gastaut syndrome · 2012-12-07 · Not FDA Approved for Orphan Indication
  • EMA fenfluramine hydrochloride (Fintepla)Treatment of Lennox-Gastaut syndrome · 27/02/2017 · PositiveEMA designation
  • EMA cannabidiol (Epidyolex)Treatment of Lennox-Gastaut syndrome · 20/03/2017 · PositiveEMA designation
  • EMA soticlestatTreatment of Lennox-Gastaut syndrome · 12/11/2021 · PositiveEMA designation

Sources: FDA OOPD · EMA orphan designations

Open Targets candidates

12

Drugs / clinical candidates · MONDO_0016532

CTD chemicals (MyDisease.info)

No CTD chemical associations returned for this Mondo ID.

Literature

Is anyone studying this?

7,884

7,884 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.

7,884 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).

5,419 in the last 10 years · medium confidence · 94.9th percentile (publications denominator)

Phrase hits: 5,401 · MeSH hits: 0

Open Europe PMC search

Who's working on it?

1,152

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
    Samanta D17 papers · 2026

    Division of Child Neurology, Department of Pediatrics, University of Arkansas for Medical Sciences, 1 Children's Way, Little Rock, AR 72202, USA.

    Papers in Europe PMC
  2. 02
    Dixon-Salazar T8 papers · 2026

    Lennox-Gastaut Syndrome Foundation, San Diego, CA, USA.

    Papers in Europe PMC
  3. 03
    Auvin S7 papers · 2026

    APHP, Robert Debré University Hospital, Pediatric Neurology Department, CRMR epilepsies rares, EpiCare Member, Paris, France.

    Papers in Europe PMC
  4. 04
    Striano P7 papers · 2026

    IRCCS Istituto Giannina Gaslini, Full Member of ERN EpiCARE, Genova, Italy.

    Papers in Europe PMC
  5. 05
    Coppola A6 papers · 2026

    Epilepsy Center, University Hospital Federico II, Naples, Italy.

    Papers in Europe PMC
  6. 06
    Patel AD6 papers · 2026

    Department of Pediatrics, The Ohio State University College of Medicine, Columbus, OH, USA; The Center for Clinical Excellence, Nationwide Children's Hospital, Columbus, OH, USA.

    Papers in Europe PMC
  7. 07
    Warren AEL6 papers · 2026

    Department of Neurosurgery, Mass General Brigham, Harvard Medical School, Boston, MA 02115, USA.

    Papers in Europe PMC
  8. 08
    Andrews JS5 papers · 2026

    Takeda Development Center Americas, Inc., 350 Massachusetts Avenue, Cambridge, MA, 02139, USA.

    Papers in Europe PMC
  9. 09
    Battaglia DI5 papers · 2026

    Pediatric Neurology and Psychiatric Unit, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.

    Papers in Europe PMC
  10. 10
    Di Bonaventura C5 papers · 2026

    Policlinico Umberto I, Sapienza University of Rome, Rome, Italy.

    Papers in Europe PMC

Clinical research

Is a treatment being tested?

49

interventional trials for this specific condition

49 interventional trials matched this specific condition name; 9 currently recruiting in our sample.

Data as of 11 September 2026 · last trial check 28 July 2026

49 interventional trials — more than 77.2% of diseases in the trials denominator have none at all (5501 of 7126; this disease is at the 97.1th percentile).

medium confidence · 97.1th percentile (trials denominator)

Recruiting interventional trials

From the matched ClinicalTrials.gov set

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

Observational and natural-history studies

9 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 8 · after dedupe 8 · already on CT.gov 0 · kept 0 · parent 0 · uncertain 8 · dropped 0 · fetched 2026-07-29

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 (8)

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 Lennox-Gastaut syndrome — 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.

("Lennox-Gastaut syndrome") OR ("MAPK10" OR "MAPK10 syndrome" OR "MAPK10-related")

Run this search on Europe PMC

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

"Lennox-Gastaut syndrome"

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: 49 interventional · 9 observational · 3 expanded access. Only interventional studies enter the trial headline.

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

Run this search on ClinicalTrials.gov

Synonyms dropped by stoplist: LGS

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 2026-07-26T20:02:41.136Z