PRX-102 (pegunigalsidase alfa), an experimental enzyme replacement therapy (ERT) for Fabry disease, safely led to significant improvements in kidney…
Articles by Joana Carvalho, PhD
Using a new harmless version of an adeno-associated virus (AAV), scientists successfully delivered a human version of alpha-galactosidase A —…
Physicians should consider Fabry disease when trying to determine the cause of recurrent episodes of fever in a patient that…
In times of emergency, telemedicine, home-based treatments, and lab tests should be adopted whenever possible to help people with…
Investigators have identified a new mutation in the GLA gene that caused Fabry disease in a 25-year-old man…
A case study of two cousins with Fabry disease who started producing high levels of neutralizing antibodies after…
A single administration of AMT-190, uniQure’s investigational gene therapy for Fabry disease, significantly increases the activity…
Treatment with PRX-102 (pegunigalsidase alfa), Protalix BioTherapeutics’ investigational enzyme replacement therapy (ERT) for Fabry disease, led…
Protalix BioTherapeutics and its partner Chiesi Global Rare Diseases, have submitted an application to the U.S. Food…
High-risk screenings for Fabry disease among individuals with chronic kidney disease (CKD) should be based on biomarkers other…