Enzyme replacement therapy: efficacy and limitations

• मुख्य लेखकों: Daniela Concolino (लेखक), Federica Deodato (लेखक), Rossella Parini (लेखक)
• स्वरूप: पुस्तक
• प्रकाशित: BMC, 2018-11-01T00:00:00Z.
• विषय: article
• ऑनलाइन पहुंच: Connect to this object online.

Abstract Enzyme replacement therapy (ERT) is available for mucopolysaccharidosis (MPS) I, MPS II, MPS VI, and MPS IVA. The efficacy of ERT has been evaluated in clinical trials and in many post-marketing studies with a long-term follow-up for MPS I, MPS II, and MPS VI. While ERT is effective in redu...