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 ...

In early trial data, INZ-701 was associated with several clinical benefits including a reduction or stabilization of carotid intima-media thickness. The Food and Drug Administration (FDA) has granted ...

BUFFALO, N.Y. – A University at Buffalo researcher working on Krabbe disease (KD) has been awarded a $250,000 grant from the Rosenau Family Research Foundation to investigate how an enzyme deficiency ...

Very long-chain acyl-CoA dehydrogenase deficiency (VLCAD), a rare inherited genetic condition, is caused by mutations in the ACADVL gene. It is associated with a disorder in fatty acid metabolism and ...

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 ...

A UB researcher working on Krabbe disease (KD) has been awarded a $250,000 grant from the Rosenau Family Research Foundation to investigate how an enzyme deficiency in the brain causes this disease, ...