Caring Cross, a 501(c)(3) non-profit dedicated to improving access to advanced therapies, and Boston Children’s Hospital have announced a collaboration to provide a sustainable, affordable pathway for ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...

Ultragenyx Pharmaceutical (RARE) has completed the rolling submission of its U.S. marketing application for its gene therapy candidate DTX401 for a rare disorder called Glycogen Storage Disease Type ...

Rice bioengineers Gang Bao and Caleb Bashor are leading a project focused on exploring how biological systems derived from nature can be engineered to deliver long-form genetic instructions for the ...

Robust, muscle-specific activation of LAMA1 following a single administration of muscle-tropic AAV vectors encoding the GNDM activator Marked improvement in survival and muscle function in DyW mouse ...

Live Science's health channel editor makes predictions about the medical breakthroughs and public health shifts to come in 2026. When you purchase through links on our site, we may earn an affiliate ...

A breakthrough study from Keck Medicine of USC may have found a powerful new triple therapy for glioblastoma, one of the deadliest brain cancers. By combining Tumor Treating Fields (TTFields), which ...