Boys with Duchenne who received Elevidys between ages 8 and 12 kept more motor function over two years than matched untreated boys, Sarepta reported at the World Muscle Society Congress. Here is what the data show, and what they don't.

A viral post claims scientists can now remove the extra chromosome behind Down syndrome. The 2025 Mie University study is real, but it was done in lab-grown cells, not babies, and it is far from a treatment. Here is the myth vs. fact.

One-year data for CTX310, a single-infusion CRISPR therapy that switches off ANGPTL3 in the liver, showed LDL cholesterol down 53% and triglycerides down 48% at the top dose, with no treatment-related serious side effects.