Sanfilippo Syndrome - MPS IIIA Investigational Drug Study

Sanfilippo syndrome Type A or mucopolysaccharidosis type IIIA (MPS IIIA) is a rare genetic disorder caused by the deficiency of the lysosomal enzyme sulfamidase. Lack of this enzyme causes buildup of large sugar molecules called glycosaminoglycans (GAGs) in the lysosomes which leads to cellular dysfunction. The buildup of GAGs causes significant damage to the body and brain. This study is a multicenter, Phase 1/2 study to assess the safety, tolerability, and efficacy of they study drug DNL126, an enzyme replacement therapy, to provide the missing enzyme to the body. Up to 28 participants with MPS IIIA will participate in the study across multiple study sites .