we propose to consider a substrate reduction therapy for MPS, which is referred to as ‘gene expression-targeted isoflavone therapy’. Apart from provision of the deficient enzyme, another ...

In this study, we report the first SDT treatment for MPS ... substrate. Rhodamine B, a nonspecific inhibitor of gag synthesis, reduced the lysosomal storage of gag in MPS cells. An in vivo therapy ...

There are no approved therapies that cross the BBB and address the central nervous system (CNS) symptoms for individuals with MPS ... replacement therapy (ERT). Concentrations of substrate in ...

Male MPS II model mice ... in greater than 95% reduction in glycosaminoglycan (GAG) substrate levels. Importantly, treatment with Sangamo's genome editing therapy at the highest dose level also ...

Heather Lau, MD, is an expert in mucopolysaccharidoses (MPS), having dedicated ... effect of AAV gene therapy. In terms of biochemical efficacy, they see a mean reduction in CSF heparan sulfate ...

a new investigational oral substrate reduction therapy for the treatment of Fabry disease. FDA's Fast Track Drug Development Program is designed to facilitate frequent interactions with the FDA ...