uniQure‘s investigational gene therapy AMT-191 markedly boosted the levels of the enzyme whose deficiency causes Fabry disease…
Articles by Steve Bryson, PhD
People with Fabry disease who develop antidrug antibodies (ADAs) against enzyme replacement therapy (ERT) tend to show higher…
A new experimental gene therapy may boost the production of alpha-galactosidase A (alpha-Gal A) — the enzyme that’s missing or…
Routine screening for gastrointestinal (GI) issues should be a standard part of clinical care for Fabry disease, according to…
Young adults with Fabry disease in Canada are at a significantly higher risk of experiencing a first-time stroke or…
A one-time infusion of gene therapy ST-920 (isaralgagene civaparvovec) improved kidney function and stabilized heart function in adults with…
Noninvasive echocardiogram imaging may serve as an early marker for left ventricular hypertrophy (LVH), a disease of the left…
In what researchers say is the first reported case of its kind, a woman in China in her 60s was…
Enzyme replacement therapy (ERT) preserved or improved heart function in two men with Fabry disease who had already…
Noninvasive in vivo confocal microscopy (IVCM) detected signs of nerve damage in the cornea — the eye’s transparent outer layer…