At a first meeting on pediatric gene and cell therapy trials, participants discussed when enrolling children before adults is ethical and when to wait.

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 ...

Peripheral nerve injuries, often caused by traumatic events such as car accidents, falls or battlefield injuries, can leave patients with long-term weakness, numbness or loss of function. Despite ...

When Harith Rajagopalan considers the millions of patients who have taken a GLP-1 drug to treat diabetes or obesity, he sees a revolution that is failing to realize its promise. “We are literally ...

Scientists have developed a new gene therapy that quiets pain at its source in the brain—without the addictive risks of opioids. Using AI to map how pain is processed, they created a targeted “off ...

ROCKVILLE, Md., July 10, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced the publication of preclinical results comparing a microdystrophin gene therapy construct that included the ...

When Domenic Catrine was born, his heel swelled up from his vitamin K shot. His circumcision site wouldn't stop bleeding. At the Children's Hospital of Philadelphia, Catrine was diagnosed as having ...

Sarepta Therapeutics is still assessing how best to prevent liver injury from its commercialized Duchenne muscular dystrophy gene therapy. But Sarepta partner Hansa Biopharma has encouraging ...

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...

RMAT designation enables intensified FDA engagement and potential accelerated pathways, implying preliminary clinical evidence for RTx-015 despite absent peer-reviewed efficacy details, endpoints, and ...

Key takeaways• Researchers at The Ohio State University College of Medicine and College of Engineering developed a novel gene ...