Mucopolysaccharidosis type IVA (MPS IVA, or Morquio syndrome type A) is an inherited
metabolic lysosomal disease caused by the deficiency of the N-acetylglucosamine-6-sulfate …

Mucopolysaccharidoses (MPS) are inborn errors of metabolism produced by a deficiency of
one of the enzymes involved in the degradation of glycosaminoglycans (GAGs). Although …

Abstract Mucopolysaccharidosis IVA (MPS IVA; Morquio A: OMIM 253000) is a lysosomal
storage disease with an autosomal recessive trait caused by the deficiency of N …

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 …

Abstract Mucopolysaccharidosis IVA (MPS IVA) is a degenerative systemic skeletal
dysplasia, in which children exhibit marked short stature and become physically …

Mucopolysaccharidosis type IVA (MPS IVA) is due to the deficiency of GALNS (N-
acetylgalactosamine 6-sulfate sulfatase) and is characterized by systemic skeletal dysplasia …

Abstract Purpose of Review The mucopolysaccharidoses (MPS) are a group of inherited
lysosomal storage disorders characterized by abnormal accumulation of …

Mucopolysaccharidosis type IVA (MPS IVA) is an autosomal congenital metabolic lysosomal
disease caused by a deficiency of the N-acetyl-galactosamine-6-sulfate sulfatase (GALNS) …

Abstract Mucopolysaccharidosis IVA (MPS IVA; Morquio syndrome) is a lysosomal storage
disorder and features systemic skeletal dysplasia that is caused by defective …

Objective To systematically review the literature and summarize the health-related quality-of-
life (HRQoL) of patients undergoing treatment for mucopolysaccharidoses (MPS), a rare …