An 8-year-old boy's life took a dramatic turn as he regained mobility through an experimental treatment for a rare genetic disorder. After months of decline, hope emerged with a pioneering drug that bypasses the genetic mutation.
Richard Schlueter, battling metastatic cancer, faces a desperate wait for experimental treatment as NIH staff cuts delay his therapy. His story sheds light on the critical impact of workforce reductions on patient care.
Robert F. Kennedy Jr. proposes expanding access to experimental medical treatments, acknowledging potential risks from unlicensed providers. Is this a step forward or a danger to public health?
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.
