KJ Muldoon was born with a devastating medical problem. Racing to solve it, doctors may have launched a 'road map' for a new type of treatment.

Get our top picks of the posters at AACR 2025 and learn more about the method behind them and the techniques used.

Axtria InsightsMAx.ai solves the "last-mile problem" in AI implementation, and seamless experimentation-to-production pipeline allow pharma companies to innovate rapidly ...

Blood creatinine-to-cystatin-c ratio, an indicator of muscle mass and strength, may be a treatment response biomarker of SMA, a study shows.

News broke yesterday that researchers in Philadelphia appear to have successfully treated a 6-month-old baby boy, called KJ, ...

A team of doctors and scientists have successfully treated a rare genetic condition with the first-ever personalized ...

The baby, KJ Muldoon of Clifton Heights, Penn., is one of 350 million people worldwide with rare diseases, most of which are ...

A research team has developed and safely delivered a personalized gene editing therapy to treat an infant with a life-threatening, incurable genetic disease. The infant, who was diagnosed with the ...

Ask scientists which gene-editing tool is most needed to advance gene therapy, and they'd probably describe a system that's ...

The UNC technology, developed by Pediatrics Professor Chengwen Li, M.D., Ph.D., uses adeno-associated viruses (AAVs) to ferry ...

A big breakthrough is being hailed in the world of gene editing, with the first custom-designed treatment using CRISPR ...