IDURONATE-2-SULFATASE REPLACEMENT THERAPY IN MPS II

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. This study examines the safety of long-term use of enzyme replacement therapy (replacing the enzyme that, when missing, causes the disease) for MPSII. If proven safe, this drug has the possibility of greatly improving the health and quality of life of all individuals affected with Mucopolysaccharidosis type II (MPS II), a disease for which there is currently no cure. He study design allows patients who successfully completed the phase I/II trial to continue to receive treatment with enzyme replacement therapy, and for patients who had received placebo in the previous trial to cross-over and receive enzyme replacement therapy.