UAB is participating in a Phase I/II trial of AMT 191, a one time gene therapy designed to enable patients with classic Fabry disease to produce their own missing enzyme, potentially reducing the need ...

Up to three in every 1,000 newborns is born with hearing loss in one or both ears. While cochlear implants have long been a life-changing option, they involve surgery and can't fully replicate the ...

Ultragenyx said that the trial of the gene therapy will continue while data from the second primary endpoint is collected.

In the 35 years since the first gene-therapy trial for a rare genetic disease, the field has advanced to new techniques and to more diseases. That first patient, 4-year-old Ashanthi DeSilva, largely ...

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 ...

Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...

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 ...

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 ...

A uniQure gene therapy slowed progression of Huntington’s disease by 75% after three years, statistically significant clinical trial results the company says will support plans for a regulatory ...