Orphan drugs in Latin America: the case of eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome
Eculizumab It has been used in the treatment of orphan diseases, such as paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), neuromyelitis optica spectrum disorder (NMOSD) and myasthenia gravis