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

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 ...

Chantez Sanford Jr., 24, of Southfield, says his life has been transformed after undergoing Lyfgenia gene therapy at Children ...

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

Nemours Children's Hospital is now offering Lyfgenia gene therapy. The treatment aims to reduce pain crises and hospital visits for young patients.

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 ...

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

After telegraphing an investigation in November, the FDA has moved to restrict the use of bluebird bio’s gene therapy Skysona in certain patients. The FDA has updated Skysona’s indication, allowing it ...

Shares of Sarepta Therapeutics SRPT rose about 2% yesterday after it announced that screening and enrollment are underway in ...

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 ...

Enh3ance trial results indicate DTX301 reduces ammonia levels by 18% and supports dietary liberalization in patients with OTC deficiency.

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