Treatment of Neuronopathic Mucopolysaccharidoses with Blood–Brain Barrier-Crossing Enzymes: Clinical Application of Receptor-Mediated Transcytosis
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DOI: 10.3390/pharmaceutics14061240
Abstract: Enzyme replacement therapy (ERT) has paved the way for treating the somatic symptoms of lysosomal storage diseases (LSDs), but the inability of intravenously administered enzymes to cross the blood–brain barrier (BBB) has left the central… read more here.
Keywords: mediated transcytosis; receptor mediated; neuronopathic mucopolysaccharidoses; blood brain ... See more keywords