エラミプレチド 化学特性,用途語,生産方法
効能
ミトコンドリア保護薬
作用機序
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.
臨床応用
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.
副作用
Side effects of elamipretide include:
Mild redness or itching at the injection site
Headache
Dizziness
エラミプレチド 上流と下流の製品情報
原材料
準備製品