Sarepta Therapeutics will cut 500 jobs and add a serious warning on the label of its muscle-disorder gene therapy Elevidys, the drugmaker said on Wednesday, following the recent deaths of two patients ...

One 7-year-old girl who received a traditional cochlear implant in one ear and the gene therapy in another, recovered almost ...

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 ...

While the CRL derails a near-term clearance, one analyst described the rejection as more of “a speed bump to approval, rather ...

Ultragenyx's UX111 gene therapy faces FDA delay due to facility issues; analysts adjust price targets amid uncertain ...

This is the first time such results have been achieved in both children and adult patients born with a specific type of ...

The FDA cited manufacturing issues but did not flag problems with Ultragenyx’s data package for UX111, with the biotech ...

It is trying to treat a devastating lifelong disease by fixing it at its source. We are able to fix the actual genetic ...

Gene therapy can improve hearing in children and adults with congenital deafness or severe hearing impairment, a new study involving researchers at Karolinska Institutet reports.

But her case is caused by a rare genetic mutation, so medications that work for 90% of people with cystic fibrosis won’t help her. The same dynamic plays out in other genetic conditions.

A team of researchers from the San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET, Milan), led by Nadia Coltella and ...

When functioning properly, this gene produces a protein that regulates the vital flow of water in and out of the lung stem cells, maintaining mucus at the right consistency. In cystic fibrosis, ...