Ionis’ ‘Breakthrough’ Alexander Disease Approval Establishes Pillar of Rare Disease Pipeline
Ionis’ ‘Breakthrough’ Alexander Disease Approval Establishes Pillar of Rare Disease Pipeline Zilganersen’s FDA approval gives families confronting Alexander disease their first disease-modifying treatment—and gives Ionis Pharmaceuticals a defining launchpad for a new generation of rare neurological medicines. For Jenny Pearson, the most frightening thing about her daughter Elise’s diagnosis was not simply the name…






