Elamipretide
La elamipretida es un péptido dirigido a las mitocondrias, en investigación para enfermedades mitocondriales y cardíacas.
Mechanism of action
Elamipretide is a cell-permeable tetrapeptide with alternating aromatic and basic residues that selectively concentrates on cardiolipin — a phospholipid found almost exclusively in the inner mitochondrial membrane that is important for cristae curvature and the organisation of the respiratory-chain complexes. By binding cardiolipin, elamipretide is proposed to stabilise cristae architecture, support electron transport and ATP production, and reduce the formation of reactive oxygen species. These mechanistic models derive largely from cell and animal models and biophysical work; the extent to which they explain clinical efficacy in humans is, given the mixed trial results, a matter of ongoing research.
La evidencia de un vistazo
Lo que muestran los estudios
Dónde discrepan los estudios
Does elamipretide have an established clinical benefit, and how does the US approval reconcile with missed endpoints in several trials?
Farmacocinética
Vías de administración en la literatura
Uso más seguro y riesgos
Efectos adversos conocidos en estudios
Interacciones y combinaciones
Riesgos e higiene en la literatura
Mixed evidence base despite accelerated approval in a niche indication
Estatus legal por país
Calculadora de reconstitución
Los péptidos vienen como polvo seco. Una vez disuelto en un líquido (reconstitución), esta calculadora responde a una única pregunta: ¿cuánta sustancia hay en un mililitro de solución después?
- 1Introduce la cantidad de sustancia del vial (figura en la etiqueta).
- 2Introduce cuánto disolvente añades.
- 3Resultado = concentración en mg por mL.
Registro de estudios
Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial
A phase 2/3 randomized clinical trial followed by an open-label extension to evaluate the effectiveness of elamipretide in Barth syndrome, a genetic disorder of mitochondrial cardiolipin metabolism
Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER
Effects of Elamipretide on Left Ventricular Function in Patients With Heart Failure With Reduced Ejection Fraction: The PROGRESS-HF Phase 2 Trial
Fuentes y metodología
Recibe nuevos estudios sobre Elamipretide
Solo ciencia, sin ventas. Cancela cuando quieras.
We store your email only for the newsletter. Unsubscribe anytime. Privacy