Companies
FDA approved Ionis ZANVASTRO (zilganersen) for Alexander disease in pediatric and adult patients.
Company 8-K AccNo 0001140361-26-035657: RNA-targeted GFAP reducer, 50 mg quarterly intrathecal; ≥5 years primary 10MWT LS mean difference 33.3% (p=0.041) at Week 61; ages 2–4 GMFM-88 improved vs control; Rare Pediatric Disease PRV awarded.