Veletri® (Epoprostenol for Injection) is intravenous prostacyclin. Unlike other epoprostenol formulations approved for PAH, Veletri® is stable at room temperature (77 F, 25 C) for up to 48 hours when administered immediately upon reconstitution and dilution, making the use of frozen gel packs unnecessary.

US residents only: www.veletri.com

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.
 

US residents only: www.4ventavis.com

Miglustat, the active ingredient of Zavesca®, is an orally available molecule with a large volume of distribution, which was developed for the treatment of type 1 Gaucher disease (GD1).
 
 

US residents only: www.zavesca.com

Digital ulcers are very painful and result in difficult-to-heal open sores that occur on fingers and toes.

Type 1 Gaucher disease, an inherited autosomal recessive disorder, is the most common disease among all inherited glycosphingolipid storage disorders, affecting approximately 7,000 patients worldwide.

Niemann-Pick type C disease is a rare genetic lysosomal storage disorder that causes severe, progressive neurological symptoms.