The FDA halted studies of two investigational gene therapies for rare pediatric diseases after a child treated in a clinical ...

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, ...

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 ...

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 ...

Scientists have discovered that blocking a key cellular enzyme thought to protect against fatty liver disease may instead increase the risk of chronic liver damage and cancer as we age.

​A new study led by researchers at the Johns Hopkins Kimmel Cancer Center, its Bloomberg~Kimmel Institute for Cancer ...

If you’ve been struggling with bloating, gas, or cramping after meals and have gone down the rabbit hole of low-FODMAP diets and elimination protocols, you might be treating the wrong problem. While ...