Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, is a devastating rare lysosomal storage disorder caused by a deficiency in the lysosomal enzyme IDS. It typically affects boys as young as 18 months. The most common symptoms include a rapid decline in learning and memory abilities, issues with heart and lung function, hyperactivity and behavioral difficulties, bone and joint deformities, and hearing loss.
The stock we are bringing to your attention today is that of REGENXBIO Inc. (RGNX), a clinical-stage biotechnology company, developing a gene therapy and one-time treatment for MPS II.
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