Invention Grant
US08765437B2 Manufacture of active highly phosphorylated human N-acetylgalactosamine-6-sulfatase and uses thereof 有权
活性高度磷酸化人N-乙酰半乳糖胺-6-硫酸酯酶的制备及其用途

Manufacture of active highly phosphorylated human N-acetylgalactosamine-6-sulfatase and uses thereof
Abstract:
This invention provides compositions of active highly phosphorylated human N-acetylgalactosamine-6-sulfatase (GALNS), and pharmaceutical compositions and formulations thereof, methods of producing and purifying GALNS, and its use in the diagnosis, prophylaxis, or treatment of diseases and conditions, including particularly lysosomal storage diseases that are caused by, or associated with, a deficiency in the GALNS enzyme, e.g., Mucopolysaccharidosis IVa (MPS IVa or Morquio A syndrome).
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