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

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-20260806-50 DOI pending Open evidence boundary →

Module significance audit · EA-20260806-50

Eight modules, one evidence-to-experiment loop.

Every entry point now declares the question it answers, its evidence unit, the object it produces, the claim it cannot support and the next legal handoff. The map also makes the DMD-only interactive surfaces explicit.

Research flow

A page is useful only when its output has a lawful next use.

No pooled score · no target winner
Data & APIVersioned schemas, provenance and checksums support every step.Machine readability does not expand the biological claim.

Eight semantic contracts

What each module means—and where interpretation must stop.

01four disease typed evidence available

Diseases

What has been measured, induced, corrected, perturbed or remains missing in each disease?

Evidence unit
Dataset-specific biological unit—donor, biopsy, clone, well or donor-derived line; individual cells are not treated as independent biological replicates by default.
Output
Typed disease summary, dataset registry, role-typed candidate objects, strict evidence states and a disease-specific next experiment.
Claim ceiling
No pooled cross-disease score, disease winner, treatment ranking, calibrated patient simulation or clinical recommendation.
Next · Choose a disease route; use the DMD interactive workflow where supported or follow the disease-specific next experiment. →
02dmd interactive gate other diseases evidence only

Questions

Can the current release answer this prespecified DMD cell-state perturbation question?

Evidence unit
One question object with disease, cell state, perturbation, time, endpoint and requested output fixed together.
Output
Answer state, available evidence layers, missing truth, claim boundary and the next discriminating experiment.
Claim ceiling
No calibrated DMD response, candidate pathway outcome, patient trajectory, efficacy estimate or clinical recommendation.
Next · Inspect the relevant context in Browse, then compare DMD candidate evidence or freeze a Study Card. →
03dmd context map and source linked records available

Browse

Which gene, cell compartment, dataset and measured context are relevant to the question?

Evidence unit
A source-linked gene, dataset or compartment record with explicit provenance and biological/statistical unit.
Output
A context map or stable evidence record that shows what is observed, contextual and missing.
Claim ceiling
No cell-specific causal effect, cell–cell interaction estimate or perturbation-conditioned DMD fate is inferred from browsing.
Next · Open a gene record, compare DMD candidates, or return to a disease module for disease-level evidence. →
04dmd evidence dimension comparison available

Gene compare

How do selected genes differ across evidence depth, source agreement, context and the experiment-blocking gap?

Evidence unit
One mapped gene record per identifier, preserving original input, mapping provenance and separate evidence dimensions.
Output
A stable comparison object and rule-traced gap-closing study suggestion without a scalar winner.
Claim ceiling
No target rank, therapeutic priority, pooled cross-disease score or silent conversion of missing evidence to zero.
Next · Send the selected evidence gap to Design and freeze the estimand, endpoint and stopping rule. →
05twenty one dmd draft study cards available

Design

What falsifiable experiment would close the named evidence gap and what result would change the decision?

Evidence unit
One versioned Study Card with estimand, endpoint, replication, QC, analysis and stopping rule.
Output
A downloadable draft design object with explicit registration gaps and links to its evidence inputs.
Claim ceiling
A draft is not a registered study, executed experiment, measured outcome, target validation or efficacy result.
Next · Inspect lifecycle state in Registry; registration and execution remain prospective. →
06bounded modelrun ledger no calibrated disease model

Models

What actually ran on a frozen task, against which baseline, with which split, metric, failure reason and permitted use?

Evidence unit
One ModelRun bound to a task, data revision, split, baseline, seed, metrics and gate decision.
Output
Model cards, a six-run execution ledger, benchmark contracts, negative results and explicit readiness gates.
Claim ceiling
Published external performance is not inherited; no calibrated DMD or multi-disease predictor is released.
Next · Carry only a frozen, gate-passing run into prospective registration and outcome comparison. →
07read only lifecycle and evidence registry available

Registry

What is draft, frozen, registered, running, measured, released, superseded or intentionally absent?

Evidence unit
One immutable versioned research object; a missing record is never interpreted as a negative result.
Output
Lifecycle states for comparisons, Study Cards, protocols, ModelRuns, predictions and outcomes, including honest zero states.
Claim ceiling
The public registry is read-only; it does not accept uploads, create persistence or promote zero outcomes into validation.
Next · Contribute a traceable outcome or evidence object, then apply the formal transition rules. →
08versioned machine readable objects available

Data & API

Which exact bytes, schema, identifier, checksum and release boundary support a displayed result?

Evidence unit
One versioned file or API object with a declared schema, provenance path and checksum identity.
Output
JSON, TSV, schemas, manifests, checksums, release bundles and a portable RO-Crate contract.
Claim ceiling
Machine readability or file availability does not expand biological claims; hosting exclusions remain declared rather than silently omitted.
Next · Verify the object, then return to the module whose scientific question it supports. →

Disease × module scope

Four diseases do not imply four identical product capabilities.

Open disease atlas →
DiseaseDiseasesQuestionsBrowseGene compareDesignModelsRegistryData & API
DMDfull typed moduleinteractive six input evidence gatedmd muscle context map and gene recordstwenty one candidate evidence comparisontwenty one draft study cardsbounded assay and dmd run ledger no calibrated modelprospective lifecycle zero registered outcomesv1 1 workbench and v2 disease objects
FSHDfull typed module with registered single cell extensionbounded evidence route no interactive predictordisease datasets and single cell state localizationnot supported no cross disease gene poolingnext experiment recommendations not frozen study cardsno calibrated disease modelreleased evidence objects no prospective outcomesv2 typed disease objects
DM1full typed module with splicing specific correctionbounded evidence route no interactive predictordisease datasets and bulk splicing evidencenot supported no cross disease gene poolingnext experiment recommendations not frozen study cardsno calibrated disease modelreleased evidence objects no prospective outcomesv2 typed disease objects
SMAfull typed module with registered single cell extensionbounded evidence route no interactive predictordisease datasets and line aware state localizationnot supported no cross disease gene poolingnext experiment recommendations not frozen study cardsno calibrated disease modelreleased evidence objects no prospective outcomesv2 typed disease objects

Reading rule: “evidence route” means a typed, bounded disease record—not an interactive predictor, a formal Study Card or a calibrated model.

Audit conclusions

The scientific boundary now travels with the navigation.