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

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

Daniel Cressy says today is a life-changing day. The man in his 20s received a gene therapy transfusion Wednesday at Manning ...

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

He is the first person in the state to receive LYFGENIA, a one-time gene therapy designed to keep the red blood cells that ...

The last available hemophilia gene therapy is temporarily unavailable, according to CSL Behring.  | The company is working with regulators to ensure stable ongoing supply for the one-time gene therapy ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain disorders known as SYNGAP1-related disorders, or SRDs. Researchers were able ...

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

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

Gene therapy approaches include gene replacement, suppression, and editing, each matched to specific genetic mechanisms in hereditary hearing loss. Preclinical studies in rodent models show promise, ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...

Researchers have developed a new approach to gene therapy that leans on the common pain reliever acetaminophen to force a variety of genetic diseases into remission. A paper published in Science ...