Sangamo Therapeutics has begun seeking accelerated approval from the U.S. Food and Drug Administration (FDA) for isaralgagene…
Articles by Marisa Wexler, MS
Markers of disease-related tissue damage in the brains of Fabry disease patients treated with…
Health Canada has approved the enzyme replacement therapy Elfabrio (pegunigalsidase alfa) to treat adults with Fabry disease. “With…
Chiesi Global Rare Diseases and Protalix Biotherapeutics are asking a committee of the European Medicines Agency (EMA) to…
Enzyme replacement therapy (ERT) may help limit damage to blood vessels in the kidneys of people with Fabry…
Treatment with the experimental gene therapy AMT-191 led to increases in levels of the enzyme whose deficit causes…
Looking at blood vessels in the eye in people with Fabry disease may give clinicians new insights into problems…
Fatty deposits called myeloid bodies in urine may help diagnose Fabry disease and monitor how people with Fabry are…
Fabry disease patients showed improvements in kidney function in the year following a single dose of gene therapy…
Levels of certain molecules in the blood may help track disease activity and kidney damage in Fabry disease, a…