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Jul 15, 2026
Researchers at MD Anderson developed an extracellular vesicle-based platform that delivers full-length DMD mRNA, restoring dystrophin production and improving muscle function in preclinical Duchenne muscular dystrophy models. The study, published in Nature Biomedical Engineering, showed reduced side effects compared with viral gene therapies.
Jul 18, 2026
England will screen all newborns for SMA after Jesy Nelson's campaign. A real-world study found Zolgensma helped children with SMA achieve motor milestones, with newborn screening leading to earlier gains.
Apr 03, 2026
Recent studies reveal AI chatbots face significant challenges in medical applications, with one study showing ChatGPT Health under-triaged 51.6% of emergency cases. Cancer patients using an AI chatbot experienced 22% withdrawal rates due to usability issues, while medical researchers show cautious adoption with 40.3% reporting AI use in research.
Mar 06, 2026
The FDA has placed a partial hold on U.S. enrollment in a Phase 2 trial of del-desiran for myotonic dystrophy type 1, citing preclinical mouse data showing blood pressure drops not seen in humans, following a serious adverse event in earlier testing.
Oct 01, 2025
The FDA approved Zycubo (copper histidinate) on January 13, 2026, as the first treatment for Menkes disease in pediatric patients, following resolution of manufacturing compliance concerns that led to an initial rejection in September 2025.
