Research from Harvard Medical School Provides New Data on Mucopolysaccharidoses (Intravenous Idursulfase for the Treatment of Mucopolysaccharidosis Type II: A Systematic Literature Review).

A recent study conducted by Harvard Medical School provides new data on mucopolysaccharidoses, specifically focusing on mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome. MPS II is a rare genetic disorder caused by a deficiency in the enzyme iduronate-2-sulfatase. The study found that enzyme replacement therapy (ERT) with intravenous idursulfase showed improved short- and long-term clinical outcomes for patients with MPS II, particularly if treatment is initiated early in life. The research provides valuable insights into the efficacy and safety of ERT for different populations of patients with MPS II, informing the overall management of the disease. [Extracted from the article]