Tag: Barth syndrome

Did you know that the synthetic peptide SS-31, also known as elamipretide, can accumulate within the mitochondria of cells at concentrations up to 5,000 times higher than in other cellular areas? This remarkable selectivity is key to understanding precisely what does SS 31 do, focusing its therapeutic effects on the cell's energy-producing powerhouse.

Following the FDA's accelerated approval in September 2025, elamipretide, now known as Forzinity, is the first approved treatment for Barth syndrome. Clinical trial data indicates that treatment duration for this chronic condition extends well beyond the initial study period, with some patients safely receiving the medication for several years.

Following the accelerated FDA approval of elamipretide (SS-31) for Barth syndrome in September 2025, this novel mitochondria-targeted peptide has garnered significant attention for its role in cellular bioenergetics. SS-31's primary function is to protect and restore the function of damaged mitochondria, which are the cellular powerhouses responsible for energy production.