Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the long-term safety and efficacy of the curative treatment, in a study led by ...

A blood stem cell gene therapy co-developed by UCLA’s Dr. Donald Kohn restored immune function in 59 of 62 children with ADA-SCID, a rare and fatal immune disorder, with no serious complications ...

A gene therapy study published recently in the journal Nature Medicine showed promising results in restoring hearing in patients of all ages with genetic hearing loss and congenital deafness. The ...

What Is Itvisma, and Why Does It Matter? Itvisma (onasemnogene abeparvovec-brve) is a gene therapy that the FDA has approved to treat spinal muscular atrophy (SMA) in adults, teens, and children 2 or ...

Youth with congenital deafness treated with gene therapy showed better progress on certain hearing measures than counterparts treated with cochlear implantation in a cohort study from China. Gene ...

Gene therapy has allowed several children born with inherited deafness to hear. A small study published Wednesday documents significantly restored hearing in five of six kids treated in China. On ...

Four decades have passed since cochlear implants gave infants born deaf the ability to hear. Now, gene therapy promises to restore natural hearing for those born with a rare form of deafness, and the ...

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

On Monday, the U.S. Food and Drug Administration (FDA) approved Novartis AG’s (NYSE: NVS) Itvisma (onasemnogene abeparvovec-brve) for children two years and older, teens, and adults with spinal ...

A single dose of a new formulation of the gene replacement therapy intrathecal onasemnogene abeparvovec ( OAV101 IT) improved movement ability in teens and children older than 2 years with spinal ...

Forbes contributors publish independent expert analyses and insights. William A. Haseltine, Ph.D., covers genomics and regenerative medicine Today, the world stands at a crossroads in genetic medicine ...

Scientists have made major progress in treating congenital deafness using gene therapy. This approach repairs hearing loss by fixing the root cause—a faulty gene known as OTOF. The OTOF gene creates ...