Shortened desensitization leading to a 2-year enzyme replacement therapy with elosulfase alfa

Abstract

Morquio syndrome (mucopolysaccharidosis type IV) is an autosomal-recessive lysosomal storage disease causing bone and joint disorders, respiratory and cardiac diseases, spinal cord compression, impaired vision, hearing loss, and dental problems.1 The only treatment is enzyme replacement therapy (ERT) with elosulfase alfa (recombinant human n-acetyl-galactosamine-6-sulfatase-110; Vimizim [5 mg/5 mL]; BioMarin Pharmaceutical Inc, London, United Kingdom), but hypersensitivity reactions (HSRs) may occur during elosulfase alfa infusion, similar to the other ERTs.