Editor’s note: On June 3, 2025, KJ Muldoon was discharged from the hospital and is now at home with his family. When a baby born in Philadelphia was announced as the first person to get a gene therapy ...

An experimental gene-editing therapy that aims to lower bad cholesterol for the long-term after a single infusion is off to a positive start in an early clinical trial. Researchers running a Phase I ...

GM1 gangliosidosis, caused by biallelic variants in GLB1, results from deficiency of lysosomal β-galactosidase, which degrades GM1 ganglioside. This fatal neurodegenerative disease currently has no ...

Abraham Lincoln, for one, may have had the syndrome—and are at very high risk of a kind of fatal rupture in the heart.

C&EN’s award-winning podcast Inflection Point leans on our 100-year archive to trace headline topics in science today back to their disparate and surprising roots. In each episode, we explore three ...