A new nonprofit wants to streamline gene therapy for diseases often avoided by pharmaceutical companies — making treatment ...

SCN2A-related developmental epileptic encephalopathy (DEE) is a rare, severe form of childhood epilepsy and one of the most ...

Kyle Muldoon tends to go with his gut. When a profound feeling came over him about this foreign concept, known as gene editing, he knew that an answer to a family crisis was in front of him. Kyle’s ...

A gene therapy designed to replace the missing protein that causes fragile X syndrome restored several disease-relevant ...

The idea behind intra-articular gene therapy for treating osteoarthritis (OA) is to deliver the gene-altering vector or cells directly to the precise site of the disease with a single injection — so ...

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 ...

Up to three in every 1,000 newborns has hearing loss in one or both ears. While cochlear implants offer remarkable hope for these children, it requires invasive surgery. These implants also cannot ...

A new gene therapy is giving people born deaf the chance to hear, often within just weeks. In a small but groundbreaking study, researchers delivered a working copy of a key hearing gene directly into ...

Rare-disease families can now invest in Mahzi Therapeutics' gene therapy, giving patients a financial stake in developing ...

The US Food and Drug Administration on Thursday approved the first gene therapy for inherited hearing loss, a one-time treatment that proved to be life-changing for a small number of children in a ...