In an infant with Barth syndrome and severe LV non-compaction cardiomyopathy, elamipretide started shortly after birth was associated with significant and sustained clinical improvement.
In plain wordsIn one infant with Barth syndrome, heart failure improved after elamipretide was started; a single case cannot show cause.
- Peptide
- Elamipretide (SS-31)
- Outcome
- IncreaseClinical heart failure improvement
- Study
n = 1
- Outcome group
- Heart function · Cardiovascular
- Studied dose
- Dose not stated
Source sentences
where ELAM was initiated shortly after birth for clinical heart failure and was associated with significant and sustained clinical improvement leading to an inactive status on the heart transplant list with eventual anticipated delisting.
1 context sentence(design, population, supporting numbers)
Barth syndrome is an exceedingly rare and potentially fatal X-linked mitochondrial disease arising from pathogenic variants in TAFAZZIN (TAZ), leading to defects in mature cardiolipin synthesis and its integration into the mitochondrial inner mitochondrial membrane.