Long-term use of Galafold (migalastat) preserved kidney function and prevented serious organ complications in Fabry disease patients over…
Articles by Lindsey Shapiro, PhD
It’s critical to conduct thorough clinical evaluations to determine whether a newly identified mutation in the GLA gene is actually causing…
Despite long-term use of enzyme replacement therapy (ERT), a man with Fabry disease in Japan had multiple strokes…
More than a third of Fabry disease patients show signs of cardiac disease progression despite use of enzyme replacement…
Researchers in England are working toward identifying and addressing barriers to reaching a Fabry disease diagnosis among underrepresented communities,…
A smartphone app, called Fabry App, for remotely assessing Fabry disease was found by patients to be helpful for…
The investigational therapy AL01211 safely lowered levels of globotriaosylceramide (Gb3) — the molecule that toxically accumulates in Fabry disease…
A year of treatment with Elfabrio (pegunigalsidase alfa) is well tolerated and slows the progression of kidney disease in…
Identifying small nerve fiber involvement early in Fabry disease (FD) could be critical to enabling timely diagnosis and treatment,…
The U.S. Food and Drug Administration (FDA) has cleared a Phase 1/2a trial to test uniQure‘s gene therapy candidate…