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US FDA grants accelerated approval to first treatment for Barth Syndrome
BioSpectrum Asia
|BioSpectrum Asia Oct 2025
The US Food and Drug Administration (FDA) has granted accelerated approval to Forzinity (elamipretide) injection as the first treatment for Barth syndrome, in patients weighing at least 30 kg.
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Barth syndrome is a rare, serious and life-threatening disease of the mitochondria (the energy-producing parts of cells). Forzinity works by binding to the inner part of the mitochondria, improving mitochon
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