Biomarker & Intervention Discovery
Pulmonary Arterial Hypertension (PAH)
Pharmacologically actionable gene targets, therapeutic agents ranked by evidence tier, and active clinical trials.
NIH funding$377MFY2025 · RCDC estimate
Funding levelSeverely Underfunded
Spontaneous remissionWithout treatment, median survival from diagnosis 2.8 years (pre-modern-therapy era); with current therapy 5-year…
Best-intervention remissionTriple upfront combination therapy (ambrisentan + tadalafil + selexipag): 41% reduction in clinical failure events…
Gap sizeLarge — most patients still receive sequential monotherapy or dual therapy rather than upfront triple therapy
Primary barrierpractice/access
Full remission profile →
Remission & chronicity
Spontaneous remission
Without treatment, median survival from diagnosis 2.8 years (pre-modern-therapy era); with current therapy 5-year survival ~50-60%; spontaneous remission essentially unknown
Best-intervention remission
Triple upfront combination therapy (ambrisentan + tadalafil + selexipag): 41% reduction in clinical failure events (TRITON trial); macitentan + tadalafil initial combination: 41% risk reduction (AMBITION); lung transplantation: 50-60% 5-year survival
Gap size
Large — most patients still receive sequential monotherapy or dual therapy rather than upfront triple therapy
Primary barrier
practice/access
Upfront combination therapy evidence is robust but drug costs (selexipag alone ~$150K/year) limit access; PAH is often missed or delayed in diagnosis (mean 2-year diagnostic delay); most patients are not treated at PAH expert centres where combination protocols are standard
Biomarker Targets & Therapeutic Agents
Gene targets queried against DGIdb, Open Targets, ChEMBL, PubMed, and Europe PMC.
Agents ranked: ● Clinical >
● Mechanistic >
● Correlative.
Pipeline results not yet available. Run: python -m biomarker_pipeline.run_for_diseases --skip-llm
Clinical Trials
Recruiting and recently completed trials from ClinicalTrials.gov.
Data retrieved 2026-07-06.
No recent clinical trials found on ClinicalTrials.gov for this condition.
Agents Found By Disease-Level Search
Searched directly from “Pulmonary Arterial Hypertension (PAH)” via ClinicalTrials.gov interventions and Open Targets'
disease→drug data — independent of the 0-gene target panel above.
This surfaces agents whose mechanism doesn't route through a curated gene (combination
therapies, standard-of-care drugs, targets outside the panel).
Not yet run. python -m biomarker_pipeline.run_disease_agent_discovery --disease "..."