mitochondrial trifunctional protein deficiency

MeSH: C566945ORPHA: 746

Overview

medical condition

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 trifunctional protein deficiency, sourced from HPO and Orphanet clinical annotations.

AreflexiaRhabdomyolysisExercise intoleranceHypotoniaLethargyMuscle weaknessFailure to thrive in infancyCongestive heart failureCardiomyopathyLeft ventricular hypertrophyHypoketotic hypoglycemiaPoor suckHypocalcemiaMuscle spasmDifficulty climbing stairsSkeletal myopathyDiffuse hepatic steatosisLower limb muscle weaknessFeeding difficulties in infancyProgressive distal muscle weaknessPeripheral neuropathyDecreased patellar reflexChronic hepatic failurePigmentary retinopathyHypoparathyroidismSeizureComaMotor delayCholestasisMitral regurgitationPes cavusRespiratory insufficiencyFrequent fallsRespiratory failureGeneralized muscle weaknessMyalgiaBabinski signTricuspid regurgitationEquinovarus deformityEquinus calcaneusArrhythmiaTip-toe gaitPrimitive reflexDistal peripheral sensory neuropathySensorimotor neuropathyRigors

Classification & Codes

MeSH Code

C566945

Orphanet Code

ORPHA:746
mitochondrial trifunctional protein deficiency
MeSHC566945
OrphanetORPHA:746
Treatments0 drug(s)
Symptoms on record46 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO