Эламипретид

Механизм действия

Elamipretide can inhibit the production of pathogenic ROS (reactive oxygen species) and restore mitochondrial ATP synthesis. Currently, the most accepted mechanism for these two actions is the direct interaction between Elamipretide and cardiolipin.

Клиническое использование

Barth syndrome is an ultra-rare genetic condition characterized by cardiac abnormalities leading to exercise intolerance, muscle weakness, debilitating fatigue, heart failure, recurrent infections, and delayed growth. There are currently no FDA- or EMA-approved therapies for patients with Barth syndrome. Elamipretide has an Orphan Drug, Fast Track and Rare Pediatric Designation from the FDA and Orphan Drug Designation from the EMA for treating Barth syndrome.

Побочные эффекты

Side effects of elamipretide include:
Mild redness or itching at the injection site
Headache
Dizziness