PRX-102 (pegunigalsidase alfa) has been approved by the U.S. Food and Drug Administration (FDA) to treat adults with…
Articles by José Lopes, PhD
The first patient in a Phase 1/2 clinical trial of Fabry disease gene therapy candidate FLT190 has been dosed.
One-time administration in mice of a gene therapy candidate known as ST-920 led to substantial increases in the activity of…
Treatment with the gene therapy candidate AVR-RD-01 showed sustained efficacy and was well-tolerated in Fabry disease patients regardless…
Treatment with  Fabrazyme (agalsidase beta) leads to greater heart and biochemical improvements in Fabry disease patients than Replagal (agalsidase alfa), a…
Two independent studies published in the journal Molecular Genetics and Metabolism and presented at the 14th Annual WORLDSymposium in San…