An application seeking approval for PRX-102 (pegunigalsidase alfa), an experimental enzyme replacement therapy for adults with Fabry disease,…
Articles by Steve Bryson, PhD
Despite disease-related eye changes, visual acuity — the sharpness of a person’s vision, with 20/20 denoting perfect clarity of…
Gene therapy ST-920 (isaralgagene civaparvovec) continues to be effective and well-tolerated in adults with Fabry disease, according to…
A man with Fabry disease undergoing long-term dialysis to support kidney function and receiving enzyme replacement therapy (ERT)…
The risk of severe COVID-19 in people with Fabry disease appears to be driven by immune system function rather…
High levels of calciprotein particles (CPP) — tiny structures that transport excess minerals in the bloodstream — were found in…
A rare case of end-stage kidney disease was discovered in a young boy with Fabry disease, a case study…
Two years of treatment with the experimental enzyme replacement therapy PRX-102 (pegunigalsidase alfa) prevented kidney decline in adults with…
Sangamo Therapeutics’ gene therapy candidate ST-920 safely and effectively increased and sustained the levels of the alpha-GalA protein,…
A recent type A meeting with the U.S. Food and Drug Administration (FDA) is likely to lead Protalix Biotherapeutics to resubmit…