Researchers reviewed how somatic mutations accumulate in normal and diseased tissues, showing that natural selection can expand clones that influence disease risk, tissue adaptation, or cellular ...

Marijuana extract shown to cut life-threatening seizures in half for children with severe epilepsy. A landmark clinical trial has revealed that a drug derived from medical marijuana can significantly ...

Mayo Clinic researchers have identified a rare mutation in the MET gene that can directly cause metabolic dysfunction-associated steatotic liver disease. The mutation disrupts the liver’s ability to ...

Researchers have discovered a new way to increase a key brain protein damaged in Rett syndrome, a rare genetic disorder that affects thousands of children worldwide. Early studies in mice and ...

The drug was finally approved in September 2025 through the FDA’s accelerated approval pathway, based on improvement in knee extensor muscle strength, for adults and children with Barth syndrome who ...

RGX-121 was rejected in early February, with Regenxbio listing the primary reasons given by the FDA as concerns about the ability to properly define a patient population, the use of a natural history ...