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 ...

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 ...

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, ...

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 ...

Do you have IBS-like flare-ups after eating sweets made with regular white sugar, starches, and other foods like oranges, bananas, and cantaloupe (when these are low FODMAP choices)? You may need to ...