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Clinical trial of gene therapy for MPS VI - a severe lysosomal storage disorder (MEUSIX)
Date du début: 1 déc. 2012, Date de fin: 30 nov. 2017,
"Mucopolysaccharidosis VI (MPS VI, or Maroteaux-Lamy syndrome; OMIM #253200) is a rare lysosomal storage disease caused by deficient activity of arylsulfatase B (ARSB). MPS VI is characterized by growth retardation, corneal clouding, cardiac valve disease, organomegaly, skeletal dysplasia, without central nervous system involvement. Thus, systemic therapies targeting peripheral organs have the pot ...
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