KOPPAKA, VISH,VELLARD, MICHEL CLAUDE,OKHAMAFE, AUGUSTUS O.,ARAYA, KIDISTI
申请号：
CA2805673
公开号：
CA2805673A1
申请日:
2011.07.22
申请国别(地区):
CA
年份:
2012
代理人:
摘要：
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).
