CORDIS Project
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This project focuses on developing a new treatment for neuronopathic Gaucher disease, which currently lacks effective therapies. It aims to advance a promising lead compound towards clinical trials to address unmet medical needs in affected patients.
Gaucher disease (GD) is an autosomal recessive disorder caused by deficient activity of acid glucosylceramidase (GBA1).
Most patients feature a non-neuronopathic (type 1) course of disease and are treated by either enzyme replacement therapy (ERT – administration of recombinant GBA1) or substrate reduction therapy (SRT – treatment with the iminosugar, Zavesca or the ceramide mimic, Eliglustat – inhibitors of glucosylceramide synthase (GCS)).
These therapeutic agents do not prevent neurological s…
UNIVERSITEIT LEIDEN
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