Heather A. Lau, MD, Assistant Professor, Department of Neurology; Associate Director, Division of Neurogenetics; Director, Lysosomal Storage Disease Program at NYU Langone Health, discusses some of the approved and emerging therapies for mucopolysaccharidoses (MPSs), a group of rare, inherited lysosomal storage disorders that are clinically characterized by abnormalities in multiple organ systems and reduced life expectancy.
Enzyme replacement therapy is currently available for five MPS disorders MPS I, MPS II, MPS IVA, MPS VI and MPS VII.
MPS I: Aldurazyme (laronidase) for patients with a confirmed diagnosis of MPS I, this therapy is available to treat the non-neurological manifestations of the disease.
MPS II: Elaprase (idursulfase) is for patients with a confirmed diagnosis of MPS II and has been approved for use in the U.S., Canada and many countries in Europe.
MPS IVA: Vimizim (elosulfase alfa) for patients with MPS IVA (Morquio A syndrome.)
MPS VI: Naglazyme (galsulfase) for individuals with a confirmed diagnosis of MPS VI.
MPS VII; MEPSEVII is a recombinant human lysosomal beta glucuronidase indicated in pediatric and adult patients for the treatment of Mucopolysaccharidosis VII (MPS VII, Sly syndrome).