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 ...

Gene therapies have ridden investor mania to huge valuations but commercialization challenges have pushed market caps to the ...

SpliceBio has closed a Series B financing round, raising $135m for the clinical development of SB-007 for Stargardt disease.

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

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

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

KJ Muldoon, a 10-month-old baby, was diagnosed with the genetic disease carbamoyl-phosphate synthetase 1 deficiency after he ...

Spacecraft unveils 1st-ever images of sun's south pole: 'Beginning of a new era' ...

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

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