Two cases of Fabry disease, diagnosed decades after symptom onset, were identified for the first time in…
Articles by Steve Bryson, PhD
An experimental genome-editing therapy safely and effectively increased levels of alpha-galactosidase A (Gal A) — which is lacking in people…
Fabrazyme (agalsidase beta) demonstrated an acceptable safety profile and resulted in sustained reductions in globotriaosylceramide…
A Phase 3 trial evaluating lucerastat, an investigational substrate reduction therapy for Fabry disease, is fully…
The first patient dosed with AVR-RD-01, an investigational gene therapy being tested in…
Fabry disease patients treated with Galafold (migalastat) had stable kidney (renal) function for up to eight and a…
A genetic screening study found a higher-than-expected prevalence of undiagnosed Fabry disease in people who had kidney failure…
Protalix BioTherapeutics and Chiesi Global Rare Diseases have launched an…
The results from a one-year extension study of the Phase 3 ATTRACT trial demonstrate the long-term safety and…
A single administration of the experimental gene therapy ST-920 in a…