Beyond The Disease, September 30, 2025

In First Drug Approved to Directly Treat Mitochondria, MitoWorld editor Gary Howard describes what resulted after years of trials and applications: the only drug approved that directly treats mitochondria. Stealth BioTherapeutics, Inc, (Stealth) received FDA approval for its drug FORZINITY™ to treat patients (over 30 kg) with Barth syndrome.

In Mitochondria Fight Pathogens by Starving Cells of Folate, MitoWorld editor Gary Howard describes a recent paper published in Science by researchers at UCLA led by Lena Pernas. They found that cells defend themselves against a pathogen by activating their mitochondria to metabolize the available folate. Without folate, the pathogen is less fit.

In Triglycerides as Brain Food, MitoWorld editor Gary Howard describes a recent paper in Nature by a multi-institute research team led by Timothy A. Ryan of Weill Cornell Medicine. Surprisingly, they found that the balance of lipid droplet metabolism is critical to the cellular energy needs at the synapse.

In Connecting Oxidative Stress and Mitochondrial Fission, MitoWorld editor Gary Howard describes a recent paper in Nature Communications in which Daria Mochly-Rosen of Stanford University discovered a small protein that facilitates the interactions of other proteins to maintain mitochondrial integrity and function during oxidative stress and that might be a therapeutic target for mitochondrial diseases.

In MitoWorld Supports Mitochondrial Disease Week, MitoWorld’s founder Gordon Freedman provides and update on the activities and features of the MitoWorld web hub in support of mitochondrial research, researchers and the clinical and patient communities, including directories, fresh content and event schedules.