Publications by authors named "Natali D Salgueiro"
Comparative pharmacokinetics and pharmacodynamics of two formulations of agalsidase beta (agalsidase Biosidus) and Fabrazyme® by intravenous infusion in healthy male volunteers.
Viridiana Berstein Eduardo M Pirotzky Hernán D Taconelli M Gabriela Gobbi Lara Beider Natali D Salgueiro
Mol Genet Metab Rep
December 2024
Fabry disease is a rare X-linked lysosomal condition that leads to the accumulation of glycosphingolipids in various tissues, causing cellular dysfunction, tissue remodeling, progressive fibrosis, and organ failure. The disease results from a deficiency in the human α-galactosidase A enzyme, responsible for breaking down glycosphingolipids like globotriaosylceramide (GL-3 or Gb3) into galactose and dihexose ceramides. In individuals diagnosed with Fabry disease, treatment from 2 years of age onwards typically involves agalsidase beta, the normal recombinant form of the defective enzyme.
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- Resistance to therapy is a significant challenge in managing cancer, particularly in luminal breast cancer where hormone and CDK4/6 inhibitors often fail.
- This study explored mechanisms of resistance using various cell lines and patient-derived cells, examining how specific kinase inhibitors affect cancer cell survival and aggressiveness.
- The findings suggest that mTOR inhibitors are more effective than PI3K inhibitors in tackling this resistance, and combining mTOR with CDK4/6 inhibitors could enhance treatment outcomes and prevent cancer recurrence.