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 ...

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 ...

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 ...

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, ...