In an early-stage clinical trial, the therapy nearly doubled the expected survival time of young patients. Still, experts caution that more research is needed. Gavin Nielsen was 2 years old when he ...

A trio of mRNA molecules could help guard against the harmful effects of aging on immune cells, a study in mice finds. A new ...

The value of a recent biochemical discovery can be seen in the case of an 8-year-old boy who was playing typical sports in August 2023, but by November needed a wheelchair because of a rare disease ...

An experimental gene therapy for high cholesterol is showing promise in clinical trials and inching closer to approval. The treatment, called VERVE-102, is being tested in people with familial ...

Patty Livermore’s father was an original 1968 Bengals season ticket holder. They attended games together, and eventually, he passed the tickets to her. Now, Livermore’s son holds the tickets. He still ...

CORONA, Calif. (KABC) -- A teenage girl is alive thanks to an experimental gene therapy treatment created at UCLA. She was born with a rare genetic disorder, so any common infection could've killed ...

What Are Clinical Trials for MASH? Clinical trials for MASH are research studies that evaluate potential new treatments for a form of liver disease called metabolic dysfunction-associated ...

A child with a rare genetic disease that affects mitochondria is the first person to receive a new experimental treatment for the potentially life-threatening condition. When you purchase through ...

Electrical stimulation of the spinal cord strengthened the muscles of three people with spinal muscular atrophy, a rare motor neuron disease. An experimental treatment that stimulates the spinal cord ...