2025 was a year of both triumph and turmoil in the medical world. While the U.S. research enterprise faced political upheaval, the relentless march of scientific progress continued unabated - delivering a host of groundbreaking innovations that promise to transform patient care. What this really means is that despite the challenges, the fundamental drivers of medical advancement - an explosion of biological data, unprecedented technological capabilities, and a surge of private investment - are stronger than ever.
From the first successful use of personalized gene therapy to treat a rare disease, to a promising new approach for slowing the progression of Huntington's, to the game-changing arrival of the first FDA-approved oral treatment for sleep apnea, 2025 was a year that showcased the transformative power of medical science. As STAT reports, "What makes 2025 unique is that these advances...came against the backdrop of government decisions that will limit even the most promising science."
Saving a Baby's Life with Gene Editing
One of the most remarkable breakthroughs of the year was the successful treatment of baby KJ Muldoon, the first person to receive personalized gene therapy for a rare disorder. Diagnosed with a life-threatening genetic condition that caused dangerous ammonia buildup, KJ became the beneficiary of a custom CRISPR-based therapy that erased his mutation and restored normal function. With a new clinical trial in the works, this approach could soon help many more individuals with rare diseases.
Slowing the March of Huntington's Disease
Another major breakthrough was the development of an experimental drug that has shown the ability to slow the progression of Huntington's disease by up to 75%. Huntington's is a devastating neurological disorder that robs patients of control over their bodies, and until now, doctors have had no effective treatments. This new therapy, which uses a virus to deliver RNA that halts the production of disease-causing proteins, represents a landmark achievement in the fight against this debilitating condition.
A New Frontier in Brain-Computer Interfaces
In a remarkable demonstration of the potential of brain-computer interface (BCI) technology, a patient with ALS became the first person in the world to control an iPad entirely by thought. Using an implantable BCI developed by Synchron, the patient was able to perform tasks like texting, browsing social media, and watching TV without using their hands or voice. As Dr. Leah Croll of Maimonides Medical Center noted, "This is really an exciting field, because I think the opportunities are boundless."
The bigger picture here is that these breakthroughs, coming against a backdrop of political turmoil, underscore the resilience and transformative potential of medical science. Despite the challenges, the pace of innovation shows no signs of slowing - and the benefits to patients are poised to be profound.