FDA grants accelerated approval for first treatment for Barth syndrome
The U.S. Food and Drug Administration has granted accelerated approval to Forzinity (elamipretide) injection as the first treatment for Barth syndrome.
Sep 25, 2025
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Congenital, Hereditary, and Neonatal Diseases and Abnormalities: News and Research on Barth Syndrome
MeSH tree: C16.131.077.121
Precision genetic target provides hope for Barth syndrome treatment
Researchers at The Hospital for Sick Children (SickKids) have uncovered a promising new therapeutic target for Barth syndrome, a rare genetic condition with no current cure.
Sep 3, 2025
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First oral drug shows promise for Barth syndrome heart and muscle symptoms
Researchers at Tohoku University have discovered that an oral drug called MA-5 can improve both heart and muscle problems in Barth syndrome, a rare genetic disorder affecting 1 in 300,000 births worldwide with no current ...
Jul 1, 2025
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Fat molecule's inability to bond with shape-shifting protein in cell's powerhouse linked to inherited metabolic disease
By studying mutations in yeast and human cells, Johns Hopkins Medicine scientists say they have found that biochemical bonds between fats and proteins in the mitochondrion, the cell's powerhouse, play a crucial role in how ...
Jun 7, 2024
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Research discovers potential new target and drug candidate for Barth syndrome
In a Nature Metabolism paper, researchers from the University of Pittsburgh detail a potential new target and a small-molecule drug candidate for treating Barth syndrome, a rare, life-threatening and currently incurable genetic ...
Nov 24, 2023
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