Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks to ...

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the most promising low-cost stocks to buy now. On February 3, Ultragenyx Pharmaceutical announced positive long-term data for UX111, which is an ...

For those born with certain types of congenital deafness, the cochlear implant has been a positive and enabling technology. It uses electronics to step in as a replacement for the biological ear that ...

All participants had adverse events, but most were moderate in severity ...

In a 15-patient, Phase 1, first-in-human trial, a one-time CRISPR-Cas9 gene-editing therapy safely reduced LDL cholesterol and triglycerides in people with difficult-to-treat lipid disorders, ...

As Eli Lilly awaits an FDA approval decision for its oral GLP-1 orforglipron, the pharma has discarded three clinical-stage ...

A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the burden of lifelong transfusions. A single one-time gene therapy could free ...

Creative Biolabs is helping researchers overcome challenges of gene therapy research by providing analytical testing ...

Gene therapy is a medical treatment that works by changing or fixing a person's genes to treat or prevent disease ...

This sponsored article was provided by a partner and is not editorial content from Los Angeles Magazine or the Engine Vision Media Network. Every cell in your body holds a unique genetic code within ...