Shares of Sarepta Therapeutics closed down as much as 42% to hit a nine-year low of $18.30 on Monday after a second death of ...

A second patient has died following treatment with Sarepta Therapeutics’ Elevidys, raising more doubts about the Duchenne muscular dystrophy (DMD) gene therapy’s safety profile. | A second patient has ...

Trump health officials signal eased FDA rules for rare disease gene therapy, but concerns linger over safety, oversight, and ...

A second patient with Duchenne muscular dystrophy has died from acute liver failure after receiving the gene therapy Elevidys ...

By Christy Santhosh and Bhanvi Satija (Reuters) -Shares of Sarepta Therapeutics slumped 36% in premarket trading on Monday after the company reported the death of a second patient in three months who ...

In comments posted in response to the Trump administration’s pharma tariff investigation, companies and industry groups ...

Oregon Health Plan (OHP) members will see increased access to lower-cost, lifesaving cell and gene therapies. The state is ...

Some doctors told him he'd live with the disease forever, but Khaled Alsheebani never stopped believing treatments would advance one day.

Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root ...

In the first webinar, Understanding the Future of Cell and Gene Therapy, Rebecca Hulinsky, MS, CGC, director, health plan genomics strategy and solutions, Quest Diagnostics, explained genetic ...

Regenxbio said Thursday that its experimental gene therapy for Duchenne muscular dystrophy was administered to two more boys and produced high levels of a miniaturized version of the protein ...