Enzyme replacement therapy (ERT) is available for mucopolysaccharidosis (MPS) I, MPS II,
MPS VI, and MPS IVA. The efficacy of ERT has been evaluated in clinical trials and in many …

The aim of this review is to summarize the evidence on efficacy, effectiveness and safety of
intravenous enzyme replacement therapy (ERT) available for mucopolysaccharidoses …

Abstract Introduction Mucopolysaccharidosis (MPS) IVA or Morquio A syndrome is an
autosomal recessive lysosomal storage disorder (LSD) caused by deficiency of the N …

Mucopolysaccharidosis VI (MPS VI) is a clinically heterogeneous and progressive disorder
with multiorgan manifestations caused by deficient N‐acetylgalactosamine‐4‐sulfatase …

Heart failure (HF) is a progressive chronic disease that remains a primary cause of death
worldwide, affecting over 64 million patients. HF can be caused by cardiomyopathies and …

Mucopolysaccharidoses (MPS) are a group of lysosomal storage disorders, which lack an
enzyme corresponding to the specific type of MPS. Enzyme replacement therapy (ERT) has …

Mucopolysaccharidosis VI (MPS VI) is a very rare autosomal recessive disorder caused by
mutations in the ARSB gene, which lead to deficient activity of the lysosomal enzyme ASB …

Research and drug developments fostered under orphan drug product development
programs have greatly assisted the introduction of efficient and safe enzyme-based …

Mucopolysaccharidosis (MPS) VI is an autosomal recessive lysosomal storage disorder
arising from deficient activity of N-acetylgalactosamine-4-sulfatase (arylsulfatase B) and …

Mucopolysaccharidoses (MPS) are a group of lysosomal storage disorders caused by a
deficiency of one of the enzymes involved in the degradation of glycosaminoglycans …