purine nucleoside phosphorylase deficiency

MeSH: C562587ORPHA: 760

Overview

combined T cell and B cell immunodeficiency that is a rare autosomal recessive metabolic disorder that has material basis in mutation in the PNP gene and characterized mainly by decreased T-cell function

Available Treatments (0)

No treatments linked yet

Clinical trials or compassionate use may be available — consult a specialist.

Clinical Presentation

Signs and symptoms associated with purine nucleoside phosphorylase deficiency, sourced from HPO and Orphanet clinical annotations.

Abnormal T cell morphologyDecreased urinary urateAbnormality of the nervous systemAutoimmune hemolytic anemiaRecurrent respiratory infectionsRecurrent infectionsAutoimmunityHypouricemiaSevere combined immunodeficiencyHumoral immunodeficiencyUnusual gastrointestinal infectionAtypical behaviorHyperactivityIntellectual disabilityAtaxiaHypotoniaSpasticityGlobal developmental delayHypertoniaDecreased total lymphocyte countSpastic paraparesisNeoplasmAbnormal central motor functionDecreased total T cell countCerebral palsySensorineural hearing impairmentStrokeAutoimmune thrombocytopeniaSystemic lupus erythematosus

Classification & Codes

MeSH Code

C562587

Orphanet Code

ORPHA:760
purine nucleoside phosphorylase deficiency
MeSHC562587
OrphanetORPHA:760
Treatments0 drug(s)
Symptoms on record29 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO