GM1-gangliosidosis and mucopolysaccharidosis type IVB (abbreviated MPS IVB, also called Morquio disease type B) are lysosomal storage diseases which are chronically debilitating and often fatal.

He participated in the phase II-III studies of Biomarin’s Vimizim, the world’s first enzymatic replacement therapy for Morquio syndrome and mucopolysaccharidosis type IV carriers.

Mucopolysaccharidosis type IV (MPS IV) is a rare disease in which the body is missing or does not have enough of an enzyme needed to break down long chains of sugar molecules.

Vimizim is indicated for the treatment of mucopolysaccharidosis, type IVA (Morquio A Syndrome, MPS IVA) in patients of all ages.

Mucopolysaccharidosis type IVB belongs to the mucopolysaccharidoses family, a group of seven inherited disorders caused by defects in lysosomal enzymes involved in the degradation of various glycosaminoglycans.