Laronidase for Genetic Disease

Genetic Disease

The FDA has approved laronidase (Aldurazyme®, BioMarin), the first treatment for certain forms of a rare genetic disease called mucopolysaccharidosis I (MPS I), which includes Hurler’s syndrome. This disease results from the absence or malfunctioning of an enzyme that breaks down molecules called glycosaminoglycans (GAG) in the cells. The buildup of GAG results in progressive cellular damage that affects appearance, physical abilities, organ function, and, in some cases, mental development.
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In one study, treated patients showed improved lung and walking capacity in a six-minute walking test after 26 weeks.

The most serious adverse drug event was an anaphylactic (allergic) reaction approximately three hours after infusion. Safety concerns related to infusion reactions included flushing, fever, headache, and rash.