Publications by authors named "Amiko Hashimoto"
Mol Genet Metab
February 2015
Article Synopsis
- - The study investigated long-term enzyme replacement therapy (ERT) for mucopolysaccharidosis IVA (MPS IVA) in mice, emphasizing treatments started at birth due to limited benefits observed in adult mice
- - Newborn mice received recombinant human GALNS through various administration routes, demonstrating enzyme distribution primarily in the liver and spleen, and some activity in bone and brain
- - Results showed that early ERT led to improved column structure in growth plates and reduced lysosomal storage in certain tissues, although some storage remained in cartilage and heart valves, highlighting the importance of starting treatment early for better outcomes.
