Its commercial products include Vimizim, an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) IV type A, a lysosomal storage disorder; Naglazyme, a recombinant form of ...

Zerlasiran was found to be well tolerated and no significant safety concerns were observed. Credit: Good dreams - Studio via ...

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.

Meeder Asset Management Inc. grew its holdings in shares of BioMarin Pharmaceutical Inc. (NASDAQ:BMRN – Free Report) by 920.8 ...

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 ...

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