| 摘要 |
PROBLEM TO BE SOLVED: To provide manufacture of highly phosphorylated active human N-acetylgalactosamine-6-sulfatase and uses thereof.SOLUTION: The invention provides production and uses of highly phosphorylated active human N-acetylgalactosamine-6-sulfatase (GALNS), pharmaceutical compositions and formulations thereof, formation and purification methods of GALNS, and, especially, uses thereof for diagnosis, prevention or treatment of diseases and conditions caused by or associated with deficiency of GALNS enzyme, including lysosomal storage diseases, for example, mucopolysaccharidosis IVa (MPSIVa or Morquio's syndrome).SELECTED DRAWING: Figure 11 |
