Latest Advances

  • Condition: Mucopolysaccharidosis VII
  • Journal: Molecular genetics and metabolism
  • Treatment Used: Vestronidase Alfa
  • Number of Patients: 12
  • Published —
This study examined the safety and effectiveness of vestronidase alfa therapy in patients with mucopolysaccharidosis VII.
  • Condition: Mucopolysaccharidosis (MPS) VII
  • Journal: International journal of molecular sciences
  • Treatment Used: Enzyme Replacement and Then Hematopoietic Stem Cell Transplantation
  • Number of Patients: 1
  • Published —
The case study followed an infant with mucopolysaccharidosis (MPS) VII and the effects of enzyme replacement (giving enzymes that the body is missing/needs) followed by hematopoietic stem cell transplantation (implanting stem cells).
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