Developing abnormal antibodies specific to glucosylsphingosine — a fat that builds up in cells of…
Articles by Ana Pena, PhD
The cough medicine ambroxol may be effective for relieving persistent bone pain in people with Gaucher disease, a…
Weakness at the connections between muscles and joints may affect bones and could contribute to the chronic fatigue felt…
Oral treatment with ambroxol — an investigational chaperone therapy — plus enzyme replacement therapy (ERT) was found…
A laboratory test using blood samples could help identify Gaucher disease (GD) patients likely to respond to experimental…
The U.S. Food and Drug Administration (FDA) granted orphan drug designation to AVR-RD-02, a gene therapy…
A scoring system of swallowing difficulties may be a simple and valuable way of staging neurological decline in infants with…
Some patients with Gaucher disease type 3c may develop non-calcified lesions in their coronary arteries, as well as tissue…
Avrobio presented updated preclinical data for AVR-RD-02, its gene therapy candidate for Gaucher disease, and announced its…
Gene activity profiles of cells from Gaucher disease patients confirm the idea that…