Michel Claude Vellard;Vish Koppaka;Augustus O. Okhamafe;Kidisti Araya
发明人:
Vish Koppaka,Michel Claude Vellard,Augustus O. Okhamafe,Kidisti Araya
申请号：
US13188907
公开号：
US08765437B2
申请日:
2011.07.22
申请国别(地区):
US
年份:
2014
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).
