Tracleer® (bosentan) is an orally active dual endothelin receptor antagonist (ERA). Tracleer® works by blocking the binding of endothelin (ET) to both ETA and ETB receptors, preventing the deleterious effects of endothelin.
Since its introduction in the USA (2001), EU (2002) and other countries worldwide, Tracleer® has become the cornerstone therapy for PAH.
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Ventavis® (inhaled Iloprost) is an inhaled formulation of iloprost, a synthetic compound that is structurally similar to prostacyclin (PGI2) – a naturally occurring molecule that causes blood vessels to dilate, limits cellular hypertrophy, and inhibits platelet aggregation.
Iloprost preferentially dilates the pulmonary arterial vascular bed.
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Miglustat, the active ingredient of Zavesca®, is an orally available molecule with a large volume of distribution, developed for the treatment of type 1 Gaucher disease.
Zavesca® has a novel mode of action as an oral inhibitor of glucosylceramide synthesis, a key enzyme involved in the biosynthesis of glycolipids. The rationale for the use of Zavesca® in type 1 Gaucher disease is to help balance overall levels of glucosylceramide by reducing its production to a level compatible with breakdown by residual enzyme activity, a unique mode of action known as “substrate reduction therapy”.
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