Its commercial products include Vimizim, an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) IV ...

Its commercial products include Vimizim, an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) IV ...

Imdusiran data from IM-PROVE I and IM-PROVE II Phase 2a clinical trials to be presented at upcoming AASLD - The Liver Meeting ...

Blood pressure was monitored twice weekly by tail-cuff plethysmography. After 21-days of DOCA-salt (n=3) and sham control (n=4), mice were perfused with 2% paraformaldehyde to quantify PNNs using the ...

The drug is the only treatment available for the debilitating and progressive condition, and works by replacing the N-acetylgalactosamine-6-sulfatase enzyme lacking in people with the disease.

To demonstrate how this can work, Andersson pointed to results from a study into the targeted delivery of a promising liver-targeted N-acetylgalactosamine (GalNAc) conjugated antisense ...

Click here to read the article now. MPS IVA is an autosomal recessive disease caused by a mutation in the N-acetylgalactosamine-5-sulfate sulfatase (GALNS) gene resulting in progressive system ...