mitochondrial complex V (ATP synthase) deficiency

ORPHA: 254913

Overview

mitochondrial metabolism disease characterized by impaired function of one or more of the proteins making up the mitochondrial proton-transporting ATP synthase complex

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 mitochondrial complex V (ATP synthase) deficiency, sourced from HPO and Orphanet clinical annotations.

SeizureAtaxiaHypotoniaMotor delayEncephalopathyMuscle weaknessHypertrophic cardiomyopathyDilated cardiomyopathyHyperammonemiaRespiratory distressLactic acidosisHyperalaninemia3-Methylglutaconic aciduriaPeripheral neuropathyArrhythmiaNeurodevelopmental delayRenal hypoplasiaHypogonadismMicrocephalySensorineural hearing impairmentStrabismusPtosisRod-cone dystrophyCataractBlindnessNystagmusOptic atrophyHypothyroidismLethargySpastic paraplegiaDysarthriaCerebellar atrophyDystoniaCerebral cortical atrophyHepatomegalyTetraplegiaExercise intoleranceShort statureMyoclonic seizureOphthalmoplegia

Classification & Codes

Orphanet Code

ORPHA:254913
mitochondrial complex V (ATP synthase) deficiency
OrphanetORPHA:254913
Treatments0 drug(s)
Symptoms on record40 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO