A 3-year-old girl who underwent a breakthrough gene therapy treatment to treat profound hearing loss can hear on her own, two ...

For children born with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), an investigational ...

New research found that gene therapy for children with a form of severe combined immunodeficiency was successful in 95% of ...

“Beyond UniQure, the pipeline includes highly promising approaches that will ideally move the goal from merely managing ...

When she was born, doctors informed Opal Sandy’s parents that their child would never hear. At the age of 11 months, she had ...

A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated ...

Miracles happen every day, everywhere, in scenarios we can hardly imagine.Although human life has become so complex that we ...

The Big Pharma’s window to take up exclusive rights to Taysha’s lead asset, dubbed TSHA-102, has officially closed, according ...

Gene therapy is linked to better nonmotor outcomes in spinal muscular atrophy type 1 compared to first-line nusinersen, new ...

In a note to investors on Sunday, analysts at Leerink Partners called these findings “compelling,” adding that they indicate ...

Elania is one of 59 children born with ADA-SCID who have been cured using this gene therapy, which will soon be submitted for ...

Long-term follow-up shows 95% success rate, no serious complications in largest ADA-SCID gene therapy study to date ...