Key Highlights
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New research shows that disease-modifying treatments for spinal muscular atrophy (SMA) type III can delay the loss of the ability to walk by an average of 12 years, with the greatest benefit seen in patients with the early-onset form (type IIIA) where the risk of losing ambulation was cut by 66%.
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This large international study of 555 individuals emphasizes that early treatment is crucial for preserving mobility, as treated patients maintained the ability to walk until a median age of 44, compared to 32 years for those who were untreated.
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A new breakthrough in cancer treatment demonstrates that a type of immunotherapy called CAR T cells can be engineered to turn on a glucose transporter (GLUT3) only when they enter a tumor, giving them a powerful energy boost to fight aggressive brain cancer (glioblastoma) more effectively.
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This “on-demand” metabolic boost helps the immune cells survive longer and work harder in the harsh tumor environment, a promising new strategy that showed significant anti-tumor effects in preclinical models and could improve the success of CAR T cell therapy for solid tumors.
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A study analyzing real-world data found that patients with hypertrophic cardiomyopathy (a thickened heart muscle) have more than double the risk of developing a serious heart valve infection (infective endocarditis) compared to the general population, with a 5-year risk of 3.25% versus 1.31%.
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This finding suggests that people with this condition may need antibiotics before dental or surgical procedures to prevent infection, a precaution that is not currently recommended by guidelines but could be a critical step in protecting their health.
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