infantile malignant osteopetrosis

ORPHA: 667

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 infantile malignant osteopetrosis, sourced from HPO and Orphanet clinical annotations.

HydrocephalusMacrocephalyHearing impairmentOtitis mediaVisual impairmentNystagmusAbnormality of visual evoked potentialsDelayed eruption of teethAbnormal rib morphologyNarrow chestAbnormal metaphysis morphologyBruising susceptibilityPallorTremorCraniosynostosisGrowth delayAbnormal pulmonary valve morphologySplenomegalyAnemiaAbnormality of metabolism/homeostasisPulmonary arterial hypertensionApneaHypophosphatemiaRecurrent respiratory infectionsHepatomegalyChronic rhinitisBone painLymphadenopathyRecurrent fracturesHypocalcemiaReduced bone mineral densityAbnormality of temperature regulationPulmonary artery stenosisAbnormal epiphysis morphologyPremature loss of primary teethBowing of the long bonesCranial nerve paralysisOptic nerve compressionAbnormal blistering of the skinOpsoclonusAbnormality of hair textureOsteopetrosisAbnormality of movement

Classification & Codes

Orphanet Code

ORPHA:667
infantile malignant osteopetrosis
OrphanetORPHA:667
Treatments0 drug(s)
Symptoms on record43 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO
infantile malignant osteopetrosis | OrphanDrug