One-Person trial tests hope for halting rare kidney disease

This study tested an artificial enzyme called ACP-501 in a single person with Familial LCAT Deficiency (FLD), a rare genetic disorder that causes very low 'good' cholesterol and often leads to kidney failure. Researchers wanted to see if regularly infusing this enzyme could improve cholesterol levels and slow or prevent the worsening of kidney problems. The participant received infusions over a year so scientists could study the treatment's effects.