Rare Disease Diagnostic Support System - Sandbox

This application provides a decision-support environment focused on a phenotypically related cluster of 11 rare disorders encompassing a total of 81 distinct phenotypic features.

The dataset is derived from eRAM (Encyclopedia of Rare Disease Annotation for Precision Medicine) by extracting the full phenotype profile of biotinidase deficiency and the set of rare diseases reported by eRAM as phenotypically similar to it. Because the source data originate from the biotinidase deficiency–centred similarity graph, phenotype overlaps between the other disorders are real but may differ slightly from those obtained by querying eRAM independently for each disease.

The system enables clinicians to:

The tool is designed to assist early recognition of rare diseases with overlapping clinical presentations, thereby facilitating diagnostic orientation prior to confirmatory genetic testing.

Diagnostic results appear at the end of the page as you select symptoms.

⬇️ Jump to results


Select the observed patient phenotypes:

Cardiovascular

Abnormality of cardiovascular system physiology
Cardiomyopathy
Cyanosis
Tachycardia


Vision / Eye

Abnormal electroretinogram
Abnormality of visual evoked potentials
Astigmatism
Cataract
High-grade hypermetropia
Iris hypopigmentation
Keratoconjunctivitis
Myopia
Nyctalopia
Nystagmus
Optic atrophy
Visual field defect
Visual loss


Hearing / Ear

Abnormality of the inner ear
Hearing impairment
Hyperacusis
Sensorineural hearing impairment
Tinnitus


Neurological / Cognitive / Psychiatric

Abnormality of the cerebellum
Anxiety
Ataxia
Cerebellar cortical atrophy
Coma
EEG abnormality
EMG abnormality
Generalized myoclonic seizures
Global developmental delay
Hallucinations
Hypertonia
Intellectual disability
Irritability
Lethargy
Muscle weakness
Muscular hypotonia
Myopathy
Psychosis
Seizures
Sleep disturbance


Skin / Hair / Dental

Abnormality of dental color
Abnormality of dental enamel
Alopecia
Carious teeth
Eczema
Exanthem
Microdontia
Perioral eczema


Respiratory

Apnea
Aspiration
Dyspnea
Hyperventilation
Increased respiratory rate or depth of breathing
Laryngeal stridor
Respiratory distress
Tachypnea


Hematologic / Systemic

Fever
Growth delay
Infertility
Low platelet count
Macrocephaly
Thrombocytopenia
Weight loss


Digestive / Metabolic / Endocrine

Anorexia
Dysphagia
Emesis
Enlarged liver
Feeding difficulties in infancy
Gastroesophageal reflux
High levels of ketone bodies
Hyperammonemia
Hypoglycemia
Metabolic acidosis
Metabolic ketoacidosis
Nausea and vomiting
Organic aciduria


Immune / Infectious

Recurrent fungal infections


Voice / Speech

Voice abnormality


Miscellaneous / Other

Spasticity


Diagnostic results will appear here as soon as you select a symptom.