LOARGYS is the first and only therapy proven to lower arginine in patients 2 years of age and older living with ARG1-D -- ARG1-D is an ultra-rare, debilitating, and progressive metabolic disease ...

Immedica Pharma today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval of Loargys® (pegzilarginase-nbln), an arginine specific enzyme indicated for the ...

DURHAM, N.C. – Many people who undergo treatment for hepatitis C develop hemolytic anemia, a disorder that destroys red blood cells. In some cases, it is so severe they have to reduce their medication ...

A Chiesi Farmaceutici drug for a rare enzyme deficiency is now approved by the FDA, making it the first U.S. treatment for a disorder that leads to a range of cognitive and muscle problems. The ...

To protect this child from the same genetic disease that killed two older siblings, treating her as soon as she was born might only work so well, the doctors knew. So they dialed back the therapeutic ...

Barbara Burton, ’73 MD, ’75 ’77 GME, professor of Pediatrics in the Division of Genetics, Birth Defects and Metabolism, was the principal investigator for this study. Currently, there are no safe or ...

In certain groups of men, glucose-6-phosphate dehydrogenase (G6PD) deficiency was linked with an increased likelihood of severe COVID-19, a Veterans Affairs cohort study found. In Black men under 65, ...