Patricia holds her Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She also served as a PhD student research assistant in the Laboratory of Doctor David A. Fidock, Department of Microbiology & Immunology, Columbia University, New York.
PRX-102 (pegunigalsidase alfa), an investigational enzyme replacement therapy (ERT) for Fabry disease, is closer to accelerated approval after a successful pre-biologics license application (BLA) meeting with the U.S. Food and ... Read more
A novel gene therapy developed by 4D Molecular Therapeutics can increase gene delivery to the heart without triggering an immune response, according to preclinical data. The gene therapy candidate ... Read more
Fabry disease shares some manifestations with rheumatic diseases, which has raised concerns of wrongful diagnosis and consequent delayed treatment. Still, patients with this rare genetic disease are unlikely to ... Read more
Protalix BioTherapeutics announced it has completed enrollment of its Phase 3 trial evaluating PRX-102 in Fabry disease patients previously treated with Shire’s Replagal (agalsidase alfa). The BRIDGE trial (NCT03018730) is an open-label switch-over study that will ... Read more
A recent review has summarized the current diagnostic tools and available treatments for Fabry disease, highlighting the challenges and potential new avenues to improve patients’ diagnoses and clinical outcomes. ... Read more
An enzyme structurally similar to alpha-galactosidase A (GLA), present in a close relative of the tobacco plant, was able to rescue Fabry disease patients’ cells. These findings suggest that plant alpha-galactosidases ... Read more
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