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, ...
What Is Loargys, and Why Does It Matter? Loargys is an enzyme-based medicine that has been granted accelerated approval by the FDA for managing high levels of the amino acid arginine (hyperargininemia ...
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 ...
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 ...
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 ...
ARG1-D is an ultra-rare, debilitating, and progressive metabolic disease resulting in persistent elevation of plasma arginine, also known as hyperargininemia FDA accelerated approval of LOARGYS is ...
An ultra-rare enzyme deficiency that keeps infants from achieving proper muscle function among other developmental delays now has its first FDA-approved treatment. The new product, a gene therapy ...
An international research team headed by Professor Dr. Thomas Dierks at Bielefeld University has discovered a hereditary enzyme deficiency. It is a subform of the mucopolysaccharidosis syndrome and ...
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