Subsequent studies by other investigators confirmed those early results, ultimately leading to the FDA approval of ...

A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists ...

Participants with Duchenne muscular dystrophy (DMD) who received RGX-202 demonstrated functional improvement and better ...

A 7-year-old from Charlotte became the first child in the area to get a new gene therapy at Atrium Health after not even being able to stand on his own a few months ago.

Panelists discuss how emerging intrathecal gene therapy for older patients with spinal muscular atrophy (SMA) shows modest ...

A combination therapy that adds a recently approved drug to the current standard of care for newly diagnosed acute myeloid ...

Health secretary Robert F. Kennedy Jr. yesterday appointed a new, smaller vaccine advisory committee at the CDC — just two ...

Leaders from the world of cell and gene therapy shared their personal stories and policy recommendations in an extraordinary ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their ...

In the first part of this series, we explored how early genetic screening and gene therapy transform the lives of newborns ...

Since the approval of Luxturna, there has been a wave of gene therapies advancing in clinical trials to treat genetic vision ...

Glioblastoma has been deadly and hard to treat, but researchers say they’ve made progress with bivalent CAR T-cell therapy ...