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

In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later, the Pfizer ...

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

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

A 24-year-old Southfield college student has become the first patient in Michigan to receive a new gene therapy that could ...

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 ...

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 ...

What Is Waskyra, and Why Does It Matter? Waskyra (etuvetidigene autotemcel) is a new gene therapy approved to treat Wiskott-Aldrich syndrome (WAS), a rare inherited condition that affects the immune ...

A new class of drugs is saving the lives of children who once had no hope. These high-tech medicines can replace defective genes. But there's a catch. Many cost millions of dollars for a single dose.

At just 24, Chantez "CJ" Sanford is quietly making Michigan medical history. He is the first person in the state to receive ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.