Enzyme Replacement Therapy (ERT) provides the missing or deficient enzyme through regular IV infusions.
Enzyme Replacement Therapy has very effectively treated Gaucher Type I for the past 20+ years. ERT has recently been developed for Fabry, certain types of Mucopolysaccharidosis (MPS) and Pompe. ERT for Niemann-Pick Types A & B is in clinical trials.
Enzymes are large molecules that do not pass the blood brain barrier into the
central nervous system. ERT is currently only effective treating the non-neurological symptoms of diseases. Efforts to by pass the blood brain barrier by injecting the enzyme directly into the spinal cord, known as intrathecal delivery, are promising in animal models and just starting in human clinical trials of MPS Type I.
Learn more:
http://www.cerezyme.com/healthcare/about/cz_hc_aboutcz.asp
