In a preclinical animal study, JCR Pharmaceuticals’ biosimilar candidate JR-051 was seen to be as effective as the approved therapy …
Articles by Marta Figueiredo, PhD
A European panel of experts has defined a set of organ-specific therapeutic goals for Fabry disease, based on a systematic…
In late-onset Fabry disease, the accumulation of a fatty molecule in cells called Gb3 — and potential cellular damage…
Electrical, structural, and functional changes in the heart, along with abnormal accumulation of fat molecules, may predict the development of cardiac…
Transplanting the liver may not be an effective strategy to restore α-galactosidase A (GLA) levels — the deficient enzyme in…
Galafold (migalastat) significantly reduces diarrhea in patients with Fabry disease and “amenable” mutations — that can respond to Galafold…
Gastrointestinal specialists should add Fabry disease to their list of possible causes of non-specific gastrointestinal symptoms, according to a review…
One of the molecules that accumulates in Fabry disease has been found to be a useful diagnostic biomarker of classic…