Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) being an oral daily medication and nusinersen (Spinraza) being administered ...
Lowering cholesterol is one of the most effective ways to reduce your risk of heart disease, and it may soon be possible to get a one-and-done gene therapy to keep cholesterol and triglyceride levels ...