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Therapeutic & Molecular Ontology

Drug, Gene & Biomarker Index

Navigate peer-reviewed breakthroughs by pharmacological class, genetic target, circulating biomarker, or diagnostic modality. Select any clinical entity to inspect all linked studies.

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Indexed Entities (24)

Gene / Target16 Peer-Reviewed Briefings

APOE4, Amyloid-β & Tau Axis

Genetic and proteopathic drivers of Alzheimer's disease and neurodegenerative cascades.

Active Specialties:NeurologyNephrologyPsychiatryCardiologyGastroenterology
Neurology editorial photograph for Cerebral Amyloid Angiopathy in Alzheimer Disease Continuum: Prevalence and Clinical Relevance in a Biomarker-Defined Memory Clinic Cohort
NEUROLOGY · REAL-WORLD COHORT

Cerebral Amyloid Angiopathy in Alzheimer Disease Continuum: Prevalence and Clinical Relevance in a Biomarker-Defined Memory Clinic Cohort

More than one-quarter of amyloid-positive patients fulfilled the Boston criteria v2.0 for probable CAA. Probable CAA was not associated...

3/5 Observational
Evidence Meter · 3/5

Real-World Observation

Finds meaningful patterns in human health records or cohorts, though observational data cannot prove direct cause.

1 min readNeurology
NEUROLOGY · CLINICAL PRACTICE

Plasma P-Tau217 as a Prognostic, Monitoring, and Risk-Stratification Biomarker of Clinical Progression in Lewy Body Disease

Plasma pTau217 is a promising prognostic, risk-stratification, and monitoring biomarker (measurable biological signal) of clinical progression in LBD, underscoring its... Although plasma phosphorylated tau-217 (pTau217) detects co-occurring Alzheimer disease (AD) neuropathologic change in Lewy body disease (LBD), relationships with long-term clinical outcomes remain unclear.

3/5 Observational
Evidence Meter · 3/5

Real-World Observation

Finds meaningful patterns in human health records or cohorts, though observational data cannot prove direct cause.

1 min readNeurology
NEUROLOGY · REAL-WORLD COHORT

Blood-Based Biomarkers as Predictors of All-Cause Dementia: A 13-Year Follow-Up in the Whitehall II Cohort Study

The long-term predictive ability of blood-based biomarkers for incident dementia in community-dwelling older adults was modest due to poor sensitivity and similar to that of a model containing known risk factors. Blood-based biomarkers are strongly correlated with amyloid positivity in research settings, but their long-term performance in heterogeneous populations remains unclear.

3/5 Observational
Evidence Meter · 3/5

Real-World Observation

Finds meaningful patterns in human health records or cohorts, though observational data cannot prove direct cause.

1 min readNeurology
NEPHROLOGY · CLINICAL PRACTICE

The macrophage paradox in renal AL amyloidosis: lessons from histology and imaging

These findings offer insights into the mechanisms of renal involvement and highlight the complex, context-dependent role of macrophages in amyloid clearance and tissue injury. In renal amyloid light chain amyloidosis, sustained and deep hematologic response drives organ response that continues to improve over time.

3/5 Observational
Evidence Meter · 3/5

Real-World Observation

Finds meaningful patterns in human health records or cohorts, though observational data cannot prove direct cause.

1 min readKidney International
NEUROLOGY · REAL-WORLD COHORT

Rapamycin Targets Brain Blood Flow in APOE4 Carriers

Low-dose rapamycin improved brain blood flow in APOE4 carriers, offering a new preventive strategy for Alzheimer's. Declining brain blood flow can precede Alzheimer's symptoms by many years, making it a critical early target for intervention.

3/5 Observational
Evidence Meter · 3/5

Real-World Observation

Finds meaningful patterns in human health records or cohorts, though observational data cannot prove direct cause.

1 min readScienceDaily
NEUROLOGY · REAL-WORLD COHORT

Deep Sleep Brain Waves Uncover Early Alzheimer's Memory Link

Disrupted deep sleep brain waves may be an early, pre-symptomatic indicator of Alzheimer's-related memory loss. This finding offers a potential new biomarker (measurable biological signal) for identifying individuals at risk for Alzheimer's disease much earlier than current clinical methods.

3/5 Observational
Evidence Meter · 3/5

Real-World Observation

Finds meaningful patterns in human health records or cohorts, though observational data cannot prove direct cause.

1 min readScienceDaily
NEUROLOGY · REAL-WORLD COHORT

Understanding apolipoprotein E as a predominant cause of Alzheimer's disease

Consequently, almost all prospective cases of Alzheimer's disease might benefit from the development of therapies to modulate APOE's role in the disease's pathophysiology. Moreover, most sporadic early-onset Alzheimer's disease might not occur without the contributions of APOE ε3 and ε4, and the two alleles may be component causes of most forms of Alzheimer's disease arising from highly penetrant genetic mutations and trisomy 21.

3/5 Observational
Evidence Meter · 3/5

Real-World Observation

Finds meaningful patterns in human health records or cohorts, though observational data cannot prove direct cause.

1 min readBrain: A Journal of Neurology
NEUROLOGY · PHASE I/II

Safety, tolerability, and efficacy of bepranemab in people living with prodromal-to-mild Alzheimer's disease: a randomised, double-blind, placebo-controlled, phase 2 trial

Treatment with bepranemab was associated with an acceptable safety profile. However, the primary endpoint was not met.

5/5 Gold Standard
Evidence Meter · 5/5

Gold-Standard Human Evidence

Backed by randomized controlled human trials or a formal meta-analysis pooling multiple clinical studies.

1 min readThe Lancet Neurology
PSYCHIATRY · RANDOMIZED CLINICAL TRIAL

Dialogical therapy plus treatment as usual versus treatment as usual in adults with distressing and persistent auditory hallucinations in the UK (Talking with Voices-II): a randomised controlled trial

TwV significantly improves personal recovery, showing efficacy (real-world effectiveness in the trial) for individuals with SMHPs who experience distressing voices. Further research comparing TwV with an active control and evaluating its implementation in routine care settings is warranted.

5/5 Gold Standard
Evidence Meter · 5/5

Gold-Standard Human Evidence

Backed by randomized controlled human trials or a formal meta-analysis pooling multiple clinical studies.

1 min readThe Lancet Psychiatry
CARDIOLOGY · PHASE III

Myocardial Responses to Eplontersen in Transthyretin Amyloid Cardiomyopathy: The CARDIO-TTRansform CMR study

Sustained transthyretin suppression with eplontersen resulted in substantially lower myocardial ECV and favorable changes in cardiac structure, biventricular systolic function, and myocardial deformation compared with placebo (inactive comparison treatment) in a cohort of patients mostly not on background stabilizer at baseline. These findings provide randomized evidence that lowering circulating transthyretin modifies myocardial disease progression in ATTR-CM and support serial multiparametric CMR for characterizing myocardial treatment response.

5/5 Gold Standard
Evidence Meter · 5/5

Gold-Standard Human Evidence

Backed by randomized controlled human trials or a formal meta-analysis pooling multiple clinical studies.

1 min readCirculation
CARDIOLOGY · RANDOMIZED CLINICAL TRIAL

Effects of Eplontersen on Cardiac Structure and Function in Patients With Transthyretin Amyloidosis With Cardiomyopathy: Insights from the CARDIO-TTRansform Trial

Among patients with TTR amyloidosis with cardiomyopathy, eplontersen modestly attenuated deterioration in prognostically important measures of LV systolic, diastolic and right ventricular function, primarily among patients not receiving stabilizers at baseline. These findings complement the clinical findings of CARDIO-TTRansform and suggest that TTR silencing may attenuate progression of cardiac dysfunction in TTR amyloidosis with cardiomyopathy.

5/5 Gold Standard
Evidence Meter · 5/5

Gold-Standard Human Evidence

Backed by randomized controlled human trials or a formal meta-analysis pooling multiple clinical studies.

1 min readCirculation
NEUROLOGY · REAL-WORLD COHORT

Understanding the Pattern of Cognitive Decline in GBA1-Related Parkinson's Disease: An Exploratory Longitudinal Multi-Cohort Study

PD GBA1 exhibits accelerated domain-generalized and more severe cognitive decline compared to PD GBA1_wildtype . Published in Annals of neurology, these peer-reviewed neurology findings provide quantitative clinical evidence to inform risk stratification, therapeutic decision-making, and follow-up validation.

4/5 Strong Trial
Evidence Meter · 4/5

Strong Clinical Evidence

Tested directly in patients through a prospective clinical trial or large multicenter human cohort.

1 min readAnnals of Neurology
NEUROLOGY · REAL-WORLD COHORT

Cerebrospinal Fluid Cytokine Expression and Relationships With Neurological Measures in Virologically Controlled People With HIV

Elevated expression of several cytokines in the CSF of virologically suppressed PWH indicates persistent immune activity and supports the existence of a central nervous system (CNS) reservoir. Numerous cytokines showed favorable associations with NP data, including "proinflammatory" cytokines such as IL-6 and TNF-α and several chemotactic cytokines, whereas some "anti-inflammatory" cytokines such as IL-10 correlated positively with unfavorable neurodegenerative markers.

3/5 Observational
Evidence Meter · 3/5

Real-World Observation

Finds meaningful patterns in human health records or cohorts, though observational data cannot prove direct cause.

1 min readAnnals of Neurology
NEUROLOGY · REAL-WORLD COHORT

Alzheimer's pathology mediates arteriolosclerosis and cerebral amyloid angiopathy associations with cognition

This study clarifies how vascular and neurodegenerative processes interact to shape cognitive impact and inform mechanistic preventions. Mediation analyses indicated that ADNC statistically accounted for about 13-29% of the associations between arteriolosclerosis and cognition across domains and 64-91% of the associations between CAA and cognition.

3/5 Observational
Evidence Meter · 3/5

Real-World Observation

Finds meaningful patterns in human health records or cohorts, though observational data cannot prove direct cause.

1 min readBrain: A Journal of Neurology
GASTROENTEROLOGY · PRECLINICAL

Single-cell landscape of alcohol-related hepatitis reveals serum amyloid a hepatocytes as key drivers of disease pathogenesis

The SAA-FPR2 pathway emerges as a potential mediator of inflammation in AH, offering potential therapeutic targets for this severe disease. This study provides new insights into the cellular heterogeneity and dynamics in ALD progression, dissecting AH at a single-cell level.

2/5 Preclinical
Evidence Meter · 2/5

Animal & Preclinical Data

Demonstrated in living animal models before large-scale human safety and dosing trials begin.

1 min readHepatology (baltimore, Md.)
NEUROLOGY · PRECLINICAL

Filamin-A links transcriptional disruption to dopaminergic decline in progressive supranuclear palsy

Together, these findings suggest that filamin-A-dependent cytoskeletal stress acts as an upstream molecular context in which immediate early gene-centred transcriptional dysregulation emerges and becomes linked to dopaminergic vulnerability in progressive supranuclear palsy. Multi-layered analyses revealed cortical monoamine depletion together with early striatal dopaminergic sensitization, indicating a close association between immediate early gene dysregulation and dopaminergic circuit imbalance.

2/5 Preclinical
Evidence Meter · 2/5

Animal & Preclinical Data

Demonstrated in living animal models before large-scale human safety and dosing trials begin.

1 min readBrain: A Journal of Neurology

Patient & Student Companion

Everyday Lab & Drug Translator

Instant plain-English guides for common blood tests and prescriptions—what normal ranges mean, what high/low values signal, and what to ask your clinician.

Lab Marker3-Month Blood Sugar

HbA1c

Measures the percentage of red blood cell hemoglobin coated with glucose over roughly 90 days.

Normal: below 5.7% · Prediabetes: 5.7%–6.4%

6.5% or higher on two tests diagnoses diabetes; very low values can occur alongside hypoglycemia or hemolytic anemia.

Ask your doctor: “Does my HbA1c match my daily fasting/post-meal glucose trends?”

Lab MarkerArtery Plaque Carrier

LDL Cholesterol

Low-density lipoprotein particles that carry cholesterol into blood vessel walls when circulating in excess.

General optimal: < 100 mg/dL (< 70 or < 55 mg/dL in high-risk hearts)

Sustained elevated LDL accelerates arterial plaque buildup; alongside ApoB, lowering LDL consistently cuts heart attack risk.

Ask your doctor: “Should we also check ApoB or Lipoprotein(a) to gauge my true plaque risk?”

Lab MarkerBone & Immune Hormone

Vitamin D (25-OH)

Blood storage marker reflecting sun-made and dietary vitamin D essential for calcium absorption and bone remodeling.

Sufficient: 20–50 ng/mL (50–125 nmol/L)

Levels under 12–20 ng/mL risk bone softening and secondary hyperparathyroidism; mega-dosing rarely beats correcting true deficiency.

Ask your doctor: “Do my labs actually show deficiency, or is a standard maintenance dose enough?”

Lab MarkerThyroid Thermostat

TSH

Pituitary hormone that signals your thyroid gland to speed up or slow down metabolism.

Typical adult range: ~0.4–4.5 mIU/L

Paradoxically, HIGH TSH usually signals an underactive thyroid (hypothyroidism), while LOW TSH points toward an overactive thyroid.

Ask your doctor: “If my TSH is borderline, should we re-test Free T4 before adjusting anything?”

Lab MarkerKidney Filter Rate

eGFR

Estimated glomerular filtration rate—how many milliliters of blood your kidneys filter every minute.

Healthy adult filter rate: 90+ mL/min/1.73m²

Values persistently below 60 for 3+ months (or elevated urine albumin) mark chronic kidney disease; single dips can simply reflect dehydration or NSAID use.

Ask your doctor: “Should we pair eGFR with a urine albumin-to-creatinine test (uACR)?”

Lab MarkerInflammation Signal

hs-CRP

High-sensitivity C-reactive protein released by the liver in response to infection, tissue stress, or vascular inflammation.

Low cardiovascular risk: < 1.0 mg/L · Elevated: > 3.0 mg/L

Mild chronic elevations flag vascular inflammation; readings above 10 mg/L usually reflect an acute cold, injury, or autoimmune flare.

Ask your doctor: “Was my CRP elevated because of a recent infection, or is it worth repeating when healthy?”

MedicationIncretin Agonists

GLP-1s

Medications (e.g., semaglutide, tirzepatide) mimicking gut satiety hormones to lower blood sugar, slow stomach emptying, and quiet brain appetite signals.

Major RCTs show 12%–22% average weight loss & 20% fewer CV events in high-risk adults

Common early effects include nausea or reflux; maintaining protein intake and resistance exercise helps preserve lean muscle mass.

Ask your doctor: “If I start a GLP-1, how do we protect lean muscle and plan long-term dosing?”

MedicationHMG-CoA Reductase Inhibitors

Statins

First-line daily medications that curb liver cholesterol synthesis and boost LDL plaque particle clearance.

Lowers LDL 30%–55% depending on dose intensity

Decades of randomized trials confirm durable reductions in heart attack, stroke, and cardiovascular death; true severe muscle injury is rare.

Ask your doctor: “What is my 10-year cardiovascular risk score, and what LDL target fits me?”

MedicationFirst-Line Metabolic Therapy

Metformin

Oldest, safest first-line type 2 diabetes drug that reduces excess liver glucose output and improves insulin sensitivity.

Lowers HbA1c by ~1.0%–1.5% without causing low blood sugar alone

Extended-release tablets cut stomach upset dramatically; long-term users should periodically monitor Vitamin B12 and kidney function (eGFR).

Ask your doctor: “Should we check my Vitamin B12 level if I have been on metformin for several years?”

MedicationSerotonin Reuptake Inhibitors

SSRIs

Medications that increase synaptic serotonin availability over 2–6 weeks to treat major depression, OCD, and anxiety disorders.

Full clinical response typically emerges at weeks 4–8

Stopping abruptly can trigger temporary brain-zap withdrawal symptoms; tapering slowly with a clinician avoids discontinuation discomfort.

Ask your doctor: “How long should we evaluate a dose before deciding whether to adjust or combine with CBT?”

SupplementCofactor Mineral

Magnesium

Essential mineral powering 300+ enzymes across nerve firing, muscle relaxation, blood pressure, and ATP energy metabolism.

Adult daily target: 310–420 mg/day from food + supplements

Standard blood serum tests miss mild tissue depletion; glycinate/citrate absorb better than oxide, while high supplemental doses can loosen stools.

Ask your doctor: “Are any of my prescription medicines lowering my magnesium stores?”