Defects in tafazzin (TAZ) are the cause of Barth syndrome (BTHS). BTHS is a severe inherited disorder, often fatal in childhood, characterized by cardiac and skeletal myopathy, short stature and neutropenia. Presentation can be slowly progressive or sudden.
Alternative names
BTHS
Cardioskeletal myopathy with neutropenia and abnormal mitochondria
3 methyl glutaconic aciduria, type II
MGA, type II
Classification
- Defects of phagocyte function
Inheritance
X-linked