Novel Enzyme Replacement Therapies for Neuropathic Mucopolysaccharidoses.

ABSTRACT: Although the advent of enzyme replacement therapy (ERT) for mucopolysaccharidoses (MPS) has paved the way for the treatment for these hereditary disorders, the blood brain barrier (BBB) has prevented patients with MPS involving the central nervous system (CNS) from benefitting from ERT. Therefore, finding ways to increase drug delivery into the brain across the BBB remains a crucial challenge for researchers and clinicians in the field. Attempts have been made to boost brain uptake of enzymes by targeting various receptors (e.g., insulin and transferrin), and several other administration routes have also been tested. This review summarizes the available information on clinical trials (completed, ongoing, and planned) of novel therapeutic agents with efficacy against CNS symptoms in neuropa

SUBMITTER: Sato Y 

PROVIDER: S-EPMC7014430 | biostudies-literature | 2020 Jan

REPOSITORIES: biostudies-literature