lysinuric protein intolerance

MeSH: C562687ORPHA: 470

Overview

Human disease

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 lysinuric protein intolerance, sourced from HPO and Orphanet clinical annotations.

Failure to thriveAbnormal renal tubule morphologyProteinuriaGlomerulonephritisNephrocalcinosisRenal tubular dysfunctionHematuriaOsteopeniaOsteoporosisIntellectual disabilityCirrhosisHepatic failureHepatosplenomegalyGrowth delayThrombocytopeniaDecreased total leukocyte countAbnormal bleedingAnemiaHyperammonemiaVomitingDiarrheaRespiratory insufficiencyHyperglycinemiaHypertriglyceridemiaHepatomegalySteatorrheaDelayed skeletal maturationElevated circulating hepatic transaminase concentrationHypercholesterolemiaIncreased LDL cholesterol concentrationHyperglutaminemiaDecreased HDL cholesterol concentrationArgininuriaHyperlysinuriaHyperalaninemiaIntraalveolar phospholipid accumulationAbnormal pulmonary interstitial morphologyHyperprolinemiaFloppy infantElevated plasma citrullineFeeding difficultiesHemophagocytosisDecreased glomerular filtration rateAbnormal circulating serine concentrationOral aversionChronic kidney diseaseIncreased circulating lactate dehydrogenase concentrationBone marrow hypercellularityCognitive impairmentHypertensionLethargyComaAbnormal heart morphologyPancreatitisRenal amyloidosisRenal tubular acidosisTubulointerstitial nephritisPulmonary fibrosisOroticaciduriaIncreased circulating ferritin concentrationOrnithinuriaAbnormality of humoral immunityMegakaryocytopeniaAbnormal circulating immunoglobulin concentrationIncreased serum zincHypofibrinogenemiaHepatic amyloidosisMembranous nephropathyAnti-dsDNA antibody positivityRenal fibrosisDecreased response to growth hormone stimulation testRecurrent bacterial infectionsPathologic fractureAntinuclear antibody positivityDecreased circulating immunoglobulin concentrationReduced circulating complement concentrationIncreased circulating immunoglobulin concentration

Classification & Codes

MeSH Code

C562687

Orphanet Code

ORPHA:470
lysinuric protein intolerance
MeSHC562687
OrphanetORPHA:470
Treatments0 drug(s)
Symptoms on record77 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO