The U.S. Food and Drug Administration has granted accelerated approval to Forzinity, the first treatment for Barth syndrome, a rare X-linked genetic disorder primarily affecting males. Developed by Stealth Biotherapeutics, the therapy targets mitochondrial dysfunction underlying the condition, offering new hope for patients with this life-threatening disease. This milestone follows years of advocacy and highlights progress in addressing ultra-rare disorders.

Targeted nanomachines for clearing senescent cells in reproductive tissues, along with gene therapies for telomerase reactivation, were reported on September 11, 2025.