Mucopolysaccharidosis type IVA (MPSIVA) or Morquio A disease, a lysosomal storage
disorder, is caused by N-acetylgalactosamine-6-sulfate sulfatase (GALNS) deficiency …

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

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 …

Gene therapy is an attractive tool for the treatment of monogenic disorders, in particular for
lysosomal storage diseases (LSD) caused by deficiencies in secretable lysosomal enzymes …

Mucopolysaccharidosis (MPS) IVA is an autosomal recessive disorder caused by deficiency
of the lysosomal enzyme N‐acetylgalatosamine‐6‐sulfate sulfatase (GALNS), which leads …

Mucopolysaccharidosis (MPS) IVA is a lysosomal storage disorder caused by mutations in
the GALNS gene that leads to the lysosomal accumulation of keratan sulfate (KS) and …

Liver gene transfer with adeno-associated viral (AAV) 2/8 vectors is being considered for
therapy of systemic diseases like mucopolysaccharidosis type VI (MPS VI), a lysosomal …

Abstract Mucopolysaccharidosis type II (MPS II; Hunter syndrome) is an X-linked inherited
disorder caused by a deficiency of the enzyme iduronate-2-sulfatase (IDS), which results in …

No treatment is available to address the unmet needs of mucopolysaccharidosis (MPS) IIIA
patients. Targeting the root cause, we developed a new self-complementary adeno …

Mucopolysaccharidosis Type IIIA (MPSIIIA) represents an unmet medical need. MPSIIIA
shares with many other lysosomal storage disorders (LSD) the characteristic of being a …