NMD-VCell Evidence & Experiment Workbench Audit disease-specific evidence, compare gaps, and freeze the missing test Module: Research Workbench · evidence-to-experiment workflow NMD = neuromuscular disorders
Search Data & API Current release

Current limit: disease modules compare measured states and intervention evidence, but none emits a calibrated patient-level prediction. DMD candidate validation remains prospective.

Research evidence only 21 prioritized candidate perturbation records 2,160 benchmark perturbation targets 250 observed human-myoblast fusion hits Prospective DMD candidate validation pending Boundary & release
21 Workbench candidates are surfaced for consideration, not ranked as validated targets 2,160 target-level perturbation responses form the frozen HepG2 benchmark substrate GSE293514 Stage B0 contributes 250 healthy-human-myoblast fusion hits and 125 individually validated genes No validated DMD perturbation prediction v1.2.0-measured-dmd-evidence Frozen 3 August 2026 Schema 1.1 Model ridge-safe-v2.3 Benchmark repeated-fold-v2.2 Build EA-20260804-49 DOI pending Open evidence boundary →

Cross-disease comparison

Compare evidence axes without naming a disease “winner.”

The table preserves different cell systems, statistical units and endpoints. Missing evidence is not converted to zero, and null findings are not hidden.

Open manifestBack to diseases
AxisDMDFSHDDM1SMA
Etiologic anchorDMDDUX4DMPK CTG repeatSMN1 / SMN2
Primary modelDMD muscle/correction + cross-context candidatesPatient myotube nuclei + inducible myoblastIsogenic myoblast + patient muscleSpinal organoid + treated patient muscle
Statistical unitDataset-specific donor / replicate1 donor/state descriptive; 4 wells/induced conditionIndependent biopsy; clone-level correctionLine-aggregated organoid; independent muscle biopsy
Strongest added axisMeasured DMD induction/correctionSparse target program + causal timing36-gene splicing convergenceConcordant KIF5A after SMN loss
Null / negative retained0/21 direct candidate DMD perturbation truthNo donor-general prevalence estimateNo global expression reversalNo global ASO expression reversal; muscle modules q>0.30
Permitted useProspective candidate experiment designState stratification and stress-intervention designMolecular/splicing correction referenceNeural response and residual-muscle study design
Claim ceilingNo calibrated DMD candidate responseNo patient efficacyNo prognosis or treatment rankingNo patient motor-outcome prediction

Measured reanalysis

The three added modules share a visual grammar, not a shared score.

Six-panel comparison of FSHD, DM1 and SMA measured evidence and intervention or correction analyses.