Clinical Application: Rare Disease

Rare Disease Diagnosis with NGS-Powered Genetic Testing

Accelerate rare disease diagnosis by shortening the diagnostic odyssey, improving diagnostic yield, and enabling precise molecular diagnoses through integrated exome and genome sequencing workflows.

ACMG-Compliant
Trio & Family Focused
Integrated CNVs
8,000+

Mendelian traits cataloged to date

100%

Integrated SNV & CNV detection workflow

~30%

Average postnatal diagnostic yield for WES

Accelerated Diagnosis

Shortening the Diagnostic Odyssey

Rare diseases, while individually rare, collectively affect millions. For many patients, the path to a diagnosis is a "diagnostic odyssey" lasting years. Next-generation sequencing (NGS) has revolutionized this field by enabling the assessment of thousands of conditions in a single assay.

Improved Diagnostic Yield

Moving beyond traditional gene panels to whole exome (WES) or whole genome (WGS) sequencing increases the probability of finding the molecular cause of disease.

Consolidated Testing

Reduce the need for sequential testing, microarray analysis, and MLPA by integrating all variant types into a single clinical pipeline.

Informed Care Decisions

A molecular diagnosis can guide medical management, inform recurrence risk for families, and connect patients to clinical trials.

Advanced Family-Based Analysis

Trio and extended family analyses are the gold standard for rare disease investigation. By comparing the proband's variants against parental genotypes, VarSeq highlights the most likely pathogenic candidates.

Trio & Quad Support

Automated filtering for de novo, compound heterozygous, and homozygous recessive patterns in trios and larger families.

Custom Pedigrees

Model complex inheritance patterns for extended families, including consanguineous pedigrees and multi-generational studies.

Segregation Analysis

Visually confirm variant segregation across affected and unaffected family members to build evidence for pathogenicity.

Mendelian Model Checks

Automated identification of variants matching expected Mendelian inheritance models (AD, AR, XL, mitochondrial).

Inheritance Filter Logic
VarSeq Template: Family-Based Filtering
De Novo FilterPassed
Proband: Het | Father: Ref | Mother: Ref
Compound Het FilterPassed
2 variants in trans: 1 Paternal | 1 Maternal
Recommended Candidate
GENE: PEX1 | CHR7:92584144 | G > A

Integrated Variant Interpretation

A complete diagnosis requires looking at the full spectrum of variation. VarSeq integrates SNVs, indels, and CNVs into a single guided ACMG workflow.

ACMG Classification

Automate the scoring of germline variants using the ACMG five-tier framework. VSClinical aggregates population data, functional predictions, and literature evidence for rapid classification.

Clinical Interpretation

Integrated CNV Scoring

First to commercialize the ACMG/ClinGen CNV guidelines. Score gains and losses alongside small variants in a unified interface, including family-based segregation analysis.

CNV Analysis

Genomic Context

Visualize variants in their genomic context with integrated GenomeBrowse. Cross-reference against master literature databases like Genomenon to find clinical evidence.

GenomeBrowse

Rare Disease Insights & Webcasts

Explore our featured articles and expert-led webcasts on clinical rare disease diagnosis.

Empower Your Rare Disease Diagnostics

Join leading clinical labs worldwide using Golden Helix to shorten the diagnostic odyssey and deliver precise molecular answers.

Trio & Family Analysis
ACMG Classification
Integrated CNV Detection