vitamin B12-unresponsive methylmalonic acidemia type mut0

ORPHA: 289916

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 vitamin B12-unresponsive methylmalonic acidemia type mut0, sourced from HPO and Orphanet clinical annotations.

Renal insufficiencyRenal tubular dysfunctionOptic atrophyIntellectual disabilityLethargyComaGlobal developmental delayHypotoniaChoreoathetosisDystoniaGrowth delayPancreatitisThrombocytopeniaDecreased total neutrophil countAnemiaHyperammonemiaNausea and vomitingChoreaRespiratory distressHepatomegalyHemiplegia/hemiparesisSepsis

Classification & Codes

Orphanet Code

ORPHA:289916
vitamin B12-unresponsive methylmalonic acidemia type mut0
OrphanetORPHA:289916
Treatments0 drug(s)
Symptoms on record22 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO