In a groundbreaking achievement, scientists have developed the first gene-editing medicine tailored for a baby suffering from a rare DNA condition. After successful treatments, KJ Muldoon is now healthier and ready to go home, showcasing a promising future for gene therapies.
Emily Kramer-Golinkoff, a cystic fibrosis patient with a rare mutation, highlights the struggles of those left behind as gene therapies show promise for others. Her journey underscores the need for equitable treatments in rare genetic conditions.
