After Treatment on Her First Day of Life, Willow’s First Steps Are a Medical Triumph

After treatment on her first day of life, Willow’s first steps are a medical triumph

Children born with a rare neuromuscular disorder are being given another chance in life thanks to a new treatment experts are calling a "game-changer".

Pediatric Gene Editing Trial for Methylmalonic Acidemia Advances

In a groundbreaking advancement that could reshape the landscape of genetic medicine, researchers have announced the successful completion of the first-ever clinical trial utilizing nuclease-free, homologous recombination-dependent gene editing in pediatric patients. This pioneering study, led by a team including Bedoyan, Morgan, and Sun, has targeted methylmalonic acidemia (MMA), a severe metabolic disorder, marking a […]