• By ICR Secretariat
  • Posted Wednesday, February 17, 2021

A gene therapy pipeline takes shape for a cluster of rare diseases

https://www.biopharmadive.com/news/gene-therapy-lysosomal-storage-disorders-fabry/594991/

The toxic globules of sugar and fat slowly pile up in the body's cells, accumulating in blood vessels and in major organs like the kidney and heart.