Lorenzo was diagnosed with an ultra-rare genetic disorder called mucopolysaccharidosis type VI (MPS VI), also known as Maroteaux-Lamy syndrome.

Mucopolysaccharidosis type VI or Maroteaux-Lamy syndrome is a rare genetic disorder caused by the deficiency of arylsulphatase B. The resultant accumulation of dermatan sulphate causes lysosomal damage.

Naglazyme is used to treat patients who have mucopolysaccharidosis VI (MPS VI or Maroteaux-Lamy syndrome).

MPS VI (Maroteaux-Lamy syndrome), is a rare pediatric genetic degenerative disease with a prevalence estimated to be 1 in 225,000 live births.

A deficiency of this enzyme is responsible for the inherited lysosomal disease, Maroteaux-Lamy syndrome (MUCOPOLYSACCHARIDOSIS VI).

A deficiency of this enzyme is responsible for the inherited lysosomal disease, Maroteaux-Lamy syndrome (MUCOPOLYSACCHARIDOSIS VI).

Like other mucopolysaccharidoses, Maroteaux-Lamy syndrome is caused by a defect in one of the enzymes that govern mucopolysaccharide metabolism, which is important in the development of connective tissue.

Inventiva is also developing a second clinical program with odiparcil (IVA 336) for the treatment of patients with mucopolysaccaridosis type VI (or Maroteaux-Lamy syndrome), a rare and severe gene disease affecting children.