An expert involved in the first successful gene therapy trial for Huntington's Disease discusses the trial results and ...

With promise to slow aging and cure some types of cancer, a new gene therapy presents cost and access hurdles for CT patients ...

Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...

The last available hemophilia gene therapy is temporarily unavailable, according to CSL Behring.  | The company is working ...

SEATTLE — Some of our most challenging health conditions result from genetic disorders like sickle cell disease and certain cancers. Gene therapy is revolutionizing how they're treated, and the Fred ...

Hemgenix for hemophilia B is temporarily unavailable, says maker CSL Behring ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their earliest stages. Today, we can intervene in the first days or weeks of life.

Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...

A new method for safely inserting large chunks of DNA into genomes has now measured up in mice, potentially paving the way ...

She has a genetic condition that affects motor nerve cells in the spinal cord, causing progressive muscle weakness. Read more ...

Solid Biosciences’ SGT-003 is the only late‑stage program to show early cardiac benefit across biomarkers and function, according to William Blair.