adenine phosphoribosyltransferase deficiency

ICD-10: E79MeSH: C538228ORPHA: 9761 Treatment Available

Overview

amino acid metabolic disorder characterized by the formation 2,8-dihydroxyadenine stones and renal failure secondary to intratubular crystalline precipitation. It has an autosomal recessive inheritance pattern

Available Treatments (1)

DrugFormStatusCountriesLead Time
allopurinol
Oral tablet, 100mg, 300mgFDA Approved, EMA Approved112d

Clinical Presentation

Signs and symptoms associated with adenine phosphoribosyltransferase deficiency, sourced from HPO and Orphanet clinical annotations.

Abnormal circulating enzyme concentration or activityRenal insufficiencyProteinuriaNephrolithiasisHypertensionAcute kidney injuryChronic kidney diseaseDysuriaRecurrent urinary tract infectionsUrinary retentionUrinary hesitancyUric acid nephrolithiasisStage 5 chronic kidney diseaseAtrial fibrillationAbdominal colicMacroscopic hematuriaFlank painOliguria

Classification & Codes

ICD-10 Code

E79

MeSH Code

C538228

Orphanet Code

ORPHA:976
adenine phosphoribosyltransferase deficiency
ICD-10E79
MeSHC538228
OrphanetORPHA:976
Treatments1 drug(s)
Symptoms on record18 signs
Statuspublished

Treatment Summary

Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO
adenine phosphoribosyltransferase deficiency | OrphanDrug