These medical breakthroughs and advances gave patients ne...
Advances delivered what may feel like medical miracles, including the first bladder transplant, a lifesaving personalized gene therapy an...
What’s Happening
Not gonna lie, Advances delivered what may feel like medical miracles, including the first bladder transplant, a lifesaving personalized gene therapy and more.
Health & Medicine These medical breakthroughs and advances gave patients new hope in 2025 Notable results include a Huntington’s treatment and custom gene therapy A gene therapy designed just for ba (center, surrounded ) saved his life. CHOP By Meghan Rosen 9 hours ago this: via email (Opens in new window) Email Click to on Facebook (Opens in new window) Facebook Click to on Reddit (Opens in new window) Reddit Click to on X (Opens in new window) X Click to print (Opens in new window) Print American science took a beating this year , with cuts to funding, jobs and credibility. (wild, right?)
And yet, scientists persisted and made progress.
The Details
These advances in 2025 spotlight how crucial it is to support biomedical research — and how much of an impact it can have on people’s lives. Slowing Huntington’s for the first time This is a brain of a person with Huntington’s disease.
The ventricles — open cavities for circulating cerebrospinal fluid — are in the center and far larger than normal, a sign of the degeneration of brain areas nearby. Zephyr/Science Source Huntington’s disease is a progressive neurological disorder that steals people’s control over their bodies.
Why This Matters
Patients may freeze up or involuntarily jerk or writhe. Doctors have no effective treatments, but an experimental drug could change that. The drug consists of a virus carrying tiny bits of RNA that halt the production of disease-causing proteins.
Scientists and researchers are watching this development closely.
Key Takeaways
- Injected into the brain, the treatment slowed Huntington’s progress 75 percent — a triumph for scientists and a promising sign for patients.
- CHOP Scientists saved a baby’s life using personalized gene therapy.
- Ba had a gene mutation that causes dangerous levels of ammonia to build up in the body.
The Bottom Line
CHOP Scientists saved a baby’s life using personalized gene therapy. Ba had a gene mutation that causes dangerous levels of ammonia to build up in the body.
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