RARE DISEASERESEARCH ATLAS
Open navigation menu

ORPHA:70589

Bronchopulmonary dysplasia

medium confidenceDisorder

Also known as: BPD

Publications

28,615

98.3th percentile

Trials

223

Interventional, condition-specific

Researchers

1,121

Distinct authors in sample

Gene link

—

Readiness

4/6

Stages with a signal

Clinical definition (Orphanet)

Bronchopulmonary is a chronic respiratory disease that results from complications related to lung injury during the treatment of infant acute respiratory distress syndrome in low-birth-weight premature infants or from abnormal lung development in older infants. Clinical signs are tachypnea, tachycardia and signs of respiratory distress such as intercostal recession, grunting and nasal flaring.

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.

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

    28,615 matched papers (18,013 in last 10 years) Source

  3. Phenotype characterisedPresent

    27 HPO annotations (e.g. Small for gestational age; Premature birth; Abnormal lung morphology) Source

  4. Animal modelNot found

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

  5. Orphan designationPresent

    6 FDA · 13 EMA designations (6 FDA orphan-indication approvals) — e.g. hydrocortisone Source

  6. Interventional trialPresent

    223 matched on ClinicalTrials.gov (37 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)

27

Associated phenotypes · MONDO:0019091

  • Small for gestational age
  • Premature birth
  • Abnormal lung morphology
  • Dyspnea
  • Emphysema

Showing 5 of 27 — 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)

19

Designations · 6 with FDA orphan-indication approval

  • FDA hydrocortisoneBronchopulmonary Dysplasia · 2020-05-13 · Not FDA Approved for Orphan Indication
  • FDA Retinol palmitateBronchopulmonary Dysplasia · 2016-08-08 · Not FDA Approved for Orphan Indication
  • FDA vitamin A palmitateBronchopulmonary Dysplasia · 2015-07-14 · Not FDA Approved for Orphan Indication
  • FDA LucinactantBronchopulmonary Dysplasia · 2006-05-23 · Not FDA Approved for Orphan Indication
  • FDA LucinactantBronchopulmonary Dysplasia · 2005-10-21 · Not FDA Approved for Orphan Indication
  • FDA Secretory leukocyte protease inhibitorBronchopulmonary Dysplasia · 1992-06-30 · Not FDA Approved for Orphan Indication
  • EMA retinol palmitatePrevention of bronchopulmonary dysplasia · 27/07/2020 · PositiveEMA designation
  • EMA recombinant fragment of human surfactant protein-DPrevention of bronchopulmonary dysplasia · 23/08/2017 · PositiveEMA designation

Sources: FDA OOPD · EMA orphan designations

Open Targets candidates

49

Drugs / clinical candidates · MONDO_0019091

CTD chemicals (MyDisease.info)

13 associated chemicals · 61 pathways. Therapeutic evidence is listed first when present — not a treatment recommendation.

  • Betamethasone · therapeutic
  • Budesonide · therapeutic
  • Calcium Gluconate · therapeutic
  • Dexamethasone · therapeutic
  • Furosemide · therapeutic
  • Glucocorticoids · therapeutic
  • Mecamylamine · therapeutic
  • Methylprednisolone · therapeutic
  • Tretinoin · therapeutic
  • Vitamin A · therapeutic
  • Indomethacin · marker/mechanism
  • Oxygen · marker/mechanism

Pathways: Antifolate resistance; MAPK signaling pathway; Cytokine-cytokine receptor interaction; NF-kappa B signaling pathway; Osteoclast differentiation; Toll-like receptor signaling pathway; NOD-like receptor signaling pathway; Cytosolic DNA-sensing pathway

MyDisease.info · MONDO:0019091

Literature

Is anyone studying this?

28,615

28,615 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.

28,615 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).

18,013 in the last 10 years · medium confidence · 98.3th percentile (publications denominator)

Phrase hits: 28,615 · MeSH hits: 1,050

Open Europe PMC search

Who's working on it?

1,121

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
    Wang X8 papers · 2026

    Department of Pediatrics, The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, China.

    Papers in Europe PMC
  2. 02
    Chen X7 papers · 2026

    Division of Surgical Research, Department of Surgery, Cooper University Hospital/Cooper Medical School of Rowan University, Camden, NJ, USA.

    Papers in Europe PMC
  3. 03
    Baraldi E5 papers · 2026

    Neonatal Intensive Care Unit and Study Centre for Preterm Birth and Bronchopulmonary Dysplasia, Department of Women's and Children's Health, University of Padua, Padua, Italy.

    Papers in Europe PMC
  4. 04
    Bonadies L5 papers · 2026

    Neonatal Intensive Care Unit and Study Centre for Preterm Birth and Bronchopulmonary Dysplasia, Department of Women's and Children's Health, University of Padua, Padua, Italy.

    Papers in Europe PMC
  5. 05
    De Luca D5 papers · 2026

    Division of Pediatrics and Neonatal Critical Care, "A. Béclère" Medical Centre, Paris Saclay University Hospital, APHP, Paris, France.

    Papers in Europe PMC
  6. 06
    Li J5 papers · 2026

    Ann & Robert H. Lurie Children's Hospital of Chicago, Stanley Manne Children's Research Institute, Chicago, IL.

    Papers in Europe PMC
  7. 07
    Onland W5 papers · 2026

    Department of Neonatology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam, The Netherlands.

    Papers in Europe PMC
  8. 08
    Wang L5 papers · 2026

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

    Papers in Europe PMC
  9. 09
    Wang S5 papers · 2026

    Department of Pediatrics, The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, China.

    Papers in Europe PMC
  10. 10
    Bhandari V4 papers · 2026

    Division of Neonatology, The Children's Regional Hospital at Cooper/Cooper Medical School of Rowan University, Camden, NJ, USA. bhandari@rowan.edu.

    Papers in Europe PMC

Clinical research

Is a treatment being tested?

223

interventional trials for this specific condition

223 interventional trials matched this specific condition name; 37 currently recruiting in our sample.

Data as of 11 September 2026

223 interventional trials — more than 77.2% of diseases in the trials denominator have none at all (5501 of 7126; this disease is at the 99.4th percentile).

medium confidence · 99.4th percentile (trials denominator)

Recruiting interventional trials

From the matched ClinicalTrials.gov set

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

Observational and natural-history studies

92 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 56 · after dedupe 56 · already on CT.gov 0 · kept 0 · parent 0 · uncertain 56 · 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 (56)

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 Bronchopulmonary dysplasia — 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.

"Bronchopulmonary dysplasia"

Run this search on Europe PMC

MeSH descriptor terms unioned into the query: Bronchopulmonary Dysplasia

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

"Bronchopulmonary dysplasia"

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

Study-type breakdown: 223 interventional · 92 observational · 0 expanded access. Only interventional studies enter the trial headline.

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

Run this search on ClinicalTrials.gov

Synonyms dropped by stoplist: BPD

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 2026-07-27T01:35:49.094Z