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 ...

The trial was small — just 12 children were treated in one or both ears — but it offers important lessons for researchers ...

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 ...

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

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

Sarepta says it has what could be the first gene therapy for limb girdle muscular dystrophy. But approval is going to be ...

UniQure's therapy, called AMT-130, reduced disease progression by 75% at 36 months in patients who received a high dose.

In a study published in Nature Medicine, Leone and her team reported the first targeted gene therapy to restore myelin, the ...

Overall and event-free survival of 100 and 95%, respectively, seen with autologous CD34+ hematopoietic stem-cell lentiviral gene therapy.

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