Barth syndrome

Barth Syndrome
Other names3-Methylglutaconic aciduria type II, X-linked cardioskeletal myopathy and neutropenia, BTHS, Cardioskeletal myopathy with neutropenia and abnormal mitochondria, Cardioskeletal myopathy-neutropenia syndrome
Cardiolipin
SpecialtyEndocrinology 
SymptomsDilated cardiomyopathy, neutropenia, short stature, muscle weakness.
ComplicationsHeart failure, delayed motor skills, infections.
Usual onsetInfancy.
CausesGenetic mutation.
PrognosisReduced life expectancy.
Frequency1-9 / 1 000 000

Barth syndrome (BTHS) is a rare but serious X-linked genetic disorder, caused by changes in phospholipid structure and metabolism. It may affect multiple body systems (though mainly characterized by pronounced pediatric-onset cardiomyopathy), and is potentially fatal. The syndrome is diagnosed almost exclusively in males.